We've found
734 Metabolic
clinical trials
Fabry Disease Clinical Trial
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Status: Enrolling,
Phase
III
Updated: 12/31/1969
Click here to add this to my saved trials
Fabry Disease Clinical Trial
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Status: Enrolling,
Phase
III
Updated: 12/31/1969
Click here to add this to my saved trials
Fabry Disease Clinical Trial
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Status: Enrolling,
Phase
III
Updated: 12/31/1969
Click here to add this to my saved trials
Hunter Syndrome Clinical Trial
Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094
Status: Enrolling,
Phase
II, III
Updated: 12/31/1969
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MPS I Clinical Trial
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
Status: Enrolling,
Phase
I
Updated: 12/31/1969
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MPS I Clinical Trial
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
Status: Enrolling,
Phase
I
Updated: 12/31/1969
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MPS I Clinical Trial
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
Status: Enrolling,
Phase
I
Updated: 12/31/1969
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Ornithine Transcarbamylase (OTC) Deficiency Clinical Trial
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency
Status: Enrolling,
Phase
I, II
Updated: 12/31/1969
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Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome Clinical Trial
Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Status: Enrolling,
Phase
I, II
Updated: 12/31/1969
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Type 1 Gaucher Disease Clinical Trial
Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease
Status: Enrolling,
Phase
IV
Updated: 12/31/1969
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Gaucher Disease Clinical Trial
Genzyme Osteopenia/Osteoporosis Study
Status: Enrolling,
Phase
Updated: 12/31/1969
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Mucopolysaccharidosis Type 3 B Clinical Trial
Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB
Status: Enrolling,
Phase
I, II
Updated: 12/31/1969
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Gaucher Disease Clinical Trial
Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
Status: Enrolling,
Phase
IV
Updated: 12/31/1969
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Gaucher Disease Clinical Trial
Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
Status: Enrolling,
Phase
IV
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Cardiology / Vascular Diseases, Endocrinology, Gastroenterology, Pharmacology / Toxicology Clinical Trial
Study of ISIS 703802 in Subjects With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Cardiology / Vascular Diseases, Endocrinology, Gastroenterology, Pharmacology / Toxicology Clinical Trial
Study of ISIS 703802 in Subjects With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Cardiology / Vascular Diseases, Endocrinology, Gastroenterology, Pharmacology / Toxicology Clinical Trial
Study of Gemcabene in Adults With FPLD
Status: Enrolling,
Phase
I, II
Updated: 4/21/2018
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Mucopolysaccharidosis Type IIIA Clinical Trial
Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
Status: Enrolling,
Phase
II, III
Updated: 12/31/1969
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Mucopolysaccharidosis Type IIIA Clinical Trial
Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
Status: Enrolling,
Phase
II, III
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Mucopolysaccharidosis Type I (MPS I) Clinical Trial
RGX-111 Gene Therapy in Patients With MPS I
Status: Enrolling,
Phase
I
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Fabry's Disease Clinical Trial
Evaluation of the Gastrointestinal Manifestation of Fabry's Disease
Status: Enrolling
Updated: 12/31/1969
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Gastroenterology, Hematology, Pharmacology / Toxicology, Other Clinical Trial
Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan
Status: Enrolling
Updated: 12/31/1969
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Oncology, Pharmacology / Toxicology Clinical Trial
Hyponatremia Study (Delayed Hyponatremia After Pituitary Surgery)
Status: Enrolling
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology Clinical Trial
X-linked Hypophosphatemia Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology Clinical Trial
X-linked Hypophosphatemia Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology Clinical Trial
X-linked Hypophosphatemia Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology Clinical Trial
Calcitriol Monotherapy for X-Linked Hypophosphatemia
Status: Enrolling,
Phase
Updated: 12/31/1969
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Cardiology / Vascular Diseases, Pharmacology / Toxicology Clinical Trial
Study of ISIS 678354 (AKCEA-APOCIII-LRx) in Patients With Hypertriglyceridemia and Established Cardiovascular Disease (CVD)
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Musculoskeletal, Pharmacology / Toxicology, Other Clinical Trial
Tolperisone in Acute Muscle Spasm of the Back
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Musculoskeletal, Pharmacology / Toxicology, Other Clinical Trial
Tolperisone in Acute Muscle Spasm of the Back
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Musculoskeletal, Pharmacology / Toxicology, Other Clinical Trial
Tolperisone in Acute Muscle Spasm of the Back
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology, Rheumatology Clinical Trial
Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
Status: Enrolling,
Phase
II
Updated: 6/23/2005
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Nephrology / Urology, Pharmacology / Toxicology Clinical Trial
Randomized Study of Folic Acid Therapy for Hyperhomocysteinemia in Patients With End Stage Renal Disease Receiving Hemodialysis
Status: Enrolling
Updated: 6/23/2005
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Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) Clinical Trial
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) Clinical Trial
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) Clinical Trial
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Fabry Disease Clinical Trial
A Study of the Effects of Fabrazyme (Agalsidase Beta) on Mother's Lactation and on the Growth, Development and Immunologic Response of Their Infants
Status: Enrolling,
Phase
IV
Updated: 10/27/2015
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Hypertriglyceridemia Clinical Trial
Effects of Amino Acids on Regional Lipid Metabolism
Status: Enrolling
Updated: 7/20/2016
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Endocrinology, Hematology, Pharmacology / Toxicology, Other Clinical Trial
Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
Status: Enrolling
Updated: 8/10/2015
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Pharmacology / Toxicology Clinical Trial
Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
Status: Enrolling
Updated: 12/8/2014
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Ornithine Transcarbamylase Deficiency Clinical Trial
Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI
Status: Enrolling
Updated: 1/27/2014
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Pharmacology / Toxicology, Other Clinical Trial
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Pharmacology / Toxicology, Other Clinical Trial
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Hypertriglyceridemia Clinical Trial
Trial of ARI-3037MO to Reduce Triglyceride Levels in Adults With Severe (≥500 mg/dl) Hypertriglyceridemia
Status: Enrolling,
Phase
II
Updated: 8/21/2015
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Pharmacology / Toxicology, Orthopedics / Podiatry Clinical Trial
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology, Orthopedics / Podiatry Clinical Trial
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology, Orthopedics / Podiatry Clinical Trial
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology, Orthopedics / Podiatry Clinical Trial
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology, Orthopedics / Podiatry Clinical Trial
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology, Orthopedics / Podiatry Clinical Trial
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
Status: Enrolling,
Phase
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Status: Enrolling,
Phase
III
Updated: 12/31/1969
Click here to add this to my saved trials
Fabry Disease Clinical Trial
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Status: Enrolling,
Phase
III
Updated: 12/31/1969
Click here to add this to my saved trials
Mucopolysaccharidosis Type IIIB Clinical Trial
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology Clinical Trial
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
Status: Enrolling,
Phase
I
Updated: 12/31/1969
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Pharmacology / Toxicology Clinical Trial
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
Status: Enrolling,
Phase
I
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Canavan Disease Clinical Trial
A Natural History Study of Canavan Disease
Status: Enrolling,
Phase
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease
Status: Enrolling,
Phase
III
Updated: 12/31/1969
Click here to add this to my saved trials
Cardiology / Vascular Diseases, Endocrinology, Gastroenterology, Pharmacology / Toxicology Clinical Trial
Study of ISIS 703802 in Subjects With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease
Status: Enrolling,
Phase
II
Updated: 12/31/1969
Click here to add this to my saved trials
Cardiology / Vascular Diseases, Endocrinology, Gastroenterology, Pharmacology / Toxicology Clinical Trial
Study of ISIS 703802 in Subjects With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease
Status: Enrolling,
Phase
II
Updated: 12/31/1969
Click here to add this to my saved trials
Gastroenterology, Pharmacology / Toxicology, Other Clinical Trial
Ropinirole for the Treatment of Muscle Cramps in Patients With Cirrhosis
Status: Enrolling,
Phase
IV
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Pharmacology / Toxicology Clinical Trial
Mucopolysaccharidosis VII Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology Clinical Trial
Mucopolysaccharidosis VII Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Gastroenterology, Pharmacology / Toxicology Clinical Trial
X-linked Hypophosphatemia Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Gastroenterology, Pharmacology / Toxicology Clinical Trial
X-linked Hypophosphatemia Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
We've found
734 Metabolic
clinical trials
Fabry Disease Clinical Trial
Updated: 12/31/1969
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Status: Enrolling,
Phase
III
Updated: 12/31/1969
Click here to add this to my saved trials
Fabry Disease Clinical Trial
Updated: 12/31/1969
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Status: Enrolling,
Phase
III
Updated: 12/31/1969
Click here to add this to my saved trials
Fabry Disease Clinical Trial
Updated: 12/31/1969
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Status: Enrolling,
Phase
III
Updated: 12/31/1969
Click here to add this to my saved trials
Fabry Disease Clinical Trial
Updated: 12/31/1969
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Status: Enrolling,
Phase
III
Updated: 12/31/1969
Click here to add this to my saved trials
Hunter Syndrome Clinical Trial
Updated: 12/31/1969
Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094
Status: Enrolling,
Phase
II, III
Updated: 12/31/1969
Click here to add this to my saved trials
MPS I Clinical Trial
Updated: 12/31/1969
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
Status: Enrolling,
Phase
I
Updated: 12/31/1969
Click here to add this to my saved trials
MPS I Clinical Trial
Updated: 12/31/1969
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
Status: Enrolling,
Phase
I
Updated: 12/31/1969
Click here to add this to my saved trials
MPS I Clinical Trial
Updated: 12/31/1969
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
Status: Enrolling,
Phase
I
Updated: 12/31/1969
Click here to add this to my saved trials
Ornithine Transcarbamylase (OTC) Deficiency Clinical Trial
Updated: 12/31/1969
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency
Status: Enrolling,
Phase
I, II
Updated: 12/31/1969
Click here to add this to my saved trials
Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome Clinical Trial
Updated: 12/31/1969
Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Status: Enrolling,
Phase
I, II
Updated: 12/31/1969
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Type 1 Gaucher Disease Clinical Trial
Updated: 12/31/1969
Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease
Status: Enrolling,
Phase
IV
Updated: 12/31/1969
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Gaucher Disease Clinical Trial
Updated: 12/31/1969
Genzyme Osteopenia/Osteoporosis Study
Status: Enrolling,
Phase
Updated: 12/31/1969
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Mucopolysaccharidosis Type 3 B Clinical Trial
Updated: 12/31/1969
Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB
Status: Enrolling,
Phase
I, II
Updated: 12/31/1969
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Gaucher Disease Clinical Trial
Updated: 12/31/1969
Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
Status: Enrolling,
Phase
IV
Updated: 12/31/1969
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Gaucher Disease Clinical Trial
Updated: 12/31/1969
Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
Status: Enrolling,
Phase
IV
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 12/31/1969
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Cardiology / Vascular Diseases, Endocrinology, Gastroenterology, Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
Study of ISIS 703802 in Subjects With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease
Status: Enrolling,
Phase
II
Updated: 12/31/1969
Click here to add this to my saved trials
Cardiology / Vascular Diseases, Endocrinology, Gastroenterology, Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
Study of ISIS 703802 in Subjects With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Cardiology / Vascular Diseases, Endocrinology, Gastroenterology, Pharmacology / Toxicology Clinical Trial
Updated: 4/21/2018
Study of Gemcabene in Adults With FPLD
Status: Enrolling,
Phase
I, II
Updated: 4/21/2018
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Mucopolysaccharidosis Type IIIA Clinical Trial
Updated: 12/31/1969
Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
Status: Enrolling,
Phase
II, III
Updated: 12/31/1969
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Mucopolysaccharidosis Type IIIA Clinical Trial
Updated: 12/31/1969
Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
Status: Enrolling,
Phase
II, III
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 12/31/1969
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 12/31/1969
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Mucopolysaccharidosis Type I (MPS I) Clinical Trial
Updated: 12/31/1969
RGX-111 Gene Therapy in Patients With MPS I
Status: Enrolling,
Phase
I
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 12/31/1969
Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 12/31/1969
Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Fabry's Disease Clinical Trial
Updated: 12/31/1969
Evaluation of the Gastrointestinal Manifestation of Fabry's Disease
Status: Enrolling
Updated: 12/31/1969
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Gastroenterology, Hematology, Pharmacology / Toxicology, Other Clinical Trial
Updated: 12/31/1969
Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan
Status: Enrolling
Updated: 12/31/1969
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Oncology, Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
Hyponatremia Study (Delayed Hyponatremia After Pituitary Surgery)
Status: Enrolling
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
X-linked Hypophosphatemia Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
X-linked Hypophosphatemia Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
X-linked Hypophosphatemia Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
Calcitriol Monotherapy for X-Linked Hypophosphatemia
Status: Enrolling,
Phase
Updated: 12/31/1969
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Cardiology / Vascular Diseases, Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
Study of ISIS 678354 (AKCEA-APOCIII-LRx) in Patients With Hypertriglyceridemia and Established Cardiovascular Disease (CVD)
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Musculoskeletal, Pharmacology / Toxicology, Other Clinical Trial
Updated: 12/31/1969
Tolperisone in Acute Muscle Spasm of the Back
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Musculoskeletal, Pharmacology / Toxicology, Other Clinical Trial
Updated: 12/31/1969
Tolperisone in Acute Muscle Spasm of the Back
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Musculoskeletal, Pharmacology / Toxicology, Other Clinical Trial
Updated: 12/31/1969
Tolperisone in Acute Muscle Spasm of the Back
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology, Rheumatology Clinical Trial
Updated: 6/23/2005
Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
Status: Enrolling,
Phase
II
Updated: 6/23/2005
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Nephrology / Urology, Pharmacology / Toxicology Clinical Trial
Updated: 6/23/2005
Randomized Study of Folic Acid Therapy for Hyperhomocysteinemia in Patients With End Stage Renal Disease Receiving Hemodialysis
Status: Enrolling
Updated: 6/23/2005
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Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) Clinical Trial
Updated: 12/31/1969
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) Clinical Trial
Updated: 12/31/1969
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) Clinical Trial
Updated: 12/31/1969
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 10/27/2015
A Study of the Effects of Fabrazyme (Agalsidase Beta) on Mother's Lactation and on the Growth, Development and Immunologic Response of Their Infants
Status: Enrolling,
Phase
IV
Updated: 10/27/2015
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials
Endocrinology, Hematology, Immunology / Infectious Diseases, Oncology, Pharmacology / Toxicology, Reproductive Clinical Trial
Updated: 12/31/1969
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Hypertriglyceridemia Clinical Trial
Updated: 7/20/2016
Effects of Amino Acids on Regional Lipid Metabolism
Status: Enrolling
Updated: 7/20/2016
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Endocrinology, Hematology, Pharmacology / Toxicology, Other Clinical Trial
Updated: 8/10/2015
Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
Status: Enrolling
Updated: 8/10/2015
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Pharmacology / Toxicology Clinical Trial
Updated: 12/8/2014
Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
Status: Enrolling
Updated: 12/8/2014
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Ornithine Transcarbamylase Deficiency Clinical Trial
Updated: 1/27/2014
Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI
Status: Enrolling
Updated: 1/27/2014
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Pharmacology / Toxicology, Other Clinical Trial
Updated: 12/31/1969
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Pharmacology / Toxicology, Other Clinical Trial
Updated: 12/31/1969
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Hypertriglyceridemia Clinical Trial
Updated: 8/21/2015
Trial of ARI-3037MO to Reduce Triglyceride Levels in Adults With Severe (≥500 mg/dl) Hypertriglyceridemia
Status: Enrolling,
Phase
II
Updated: 8/21/2015
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Pharmacology / Toxicology, Orthopedics / Podiatry Clinical Trial
Updated: 12/31/1969
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology, Orthopedics / Podiatry Clinical Trial
Updated: 12/31/1969
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology, Orthopedics / Podiatry Clinical Trial
Updated: 12/31/1969
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology, Orthopedics / Podiatry Clinical Trial
Updated: 12/31/1969
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology, Orthopedics / Podiatry Clinical Trial
Updated: 12/31/1969
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology, Orthopedics / Podiatry Clinical Trial
Updated: 12/31/1969
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 12/31/1969
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
Status: Enrolling,
Phase
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 12/31/1969
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 12/31/1969
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Mucopolysaccharidosis Type IIIB Clinical Trial
Updated: 12/31/1969
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
Status: Enrolling,
Phase
I
Updated: 12/31/1969
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Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
Status: Enrolling,
Phase
I
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 12/31/1969
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Canavan Disease Clinical Trial
Updated: 12/31/1969
A Natural History Study of Canavan Disease
Status: Enrolling,
Phase
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 12/31/1969
Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease
Status: Enrolling,
Phase
III
Updated: 12/31/1969
Click here to add this to my saved trials
Fabry Disease Clinical Trial
Updated: 12/31/1969
Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease
Status: Enrolling,
Phase
III
Updated: 12/31/1969
Click here to add this to my saved trials
Cardiology / Vascular Diseases, Endocrinology, Gastroenterology, Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
Study of ISIS 703802 in Subjects With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease
Status: Enrolling,
Phase
II
Updated: 12/31/1969
Click here to add this to my saved trials
Cardiology / Vascular Diseases, Endocrinology, Gastroenterology, Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
Study of ISIS 703802 in Subjects With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease
Status: Enrolling,
Phase
II
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology, Other Clinical Trial
Updated: 12/31/1969
Ropinirole for the Treatment of Muscle Cramps in Patients With Cirrhosis
Status: Enrolling,
Phase
IV
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 12/31/1969
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Fabry Disease Clinical Trial
Updated: 12/31/1969
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to
Status: Enrolling,
Phase
III
Updated: 12/31/1969
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Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
Mucopolysaccharidosis VII Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
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Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
Mucopolysaccharidosis VII Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
X-linked Hypophosphatemia Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
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Gastroenterology, Pharmacology / Toxicology Clinical Trial
Updated: 12/31/1969
X-linked Hypophosphatemia Disease Monitoring Program
Status: Enrolling,
Phase
Updated: 12/31/1969
Click here to add this to my saved trials