Natural History Study of Cystathionine Beta-synthase Deficiency Homocystinuria (CBSDH)



Status:Recruiting
Conditions:Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:5 - 65
Updated:7/5/2018
Start Date:January 2017
End Date:April 2021
Contact:Anne Kuan
Email:anne.kuan@neovii.com
Phone:+1 781 966 3832

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Trial Summary
Trial Overview
Inclusion Criteria

A Multicenter, Observational, Prospective, Natural History Study of Homocystinuria Due to Cystathionine Beta-synthase Deficiency in Pediatric and Adult Patients

The purpose of the study is to characterize the clinical course of Cystathionine
Beta-synthase Deficiency Homocystinuria (CBSDH) in pediatric and adult patients under current
clinical management practices


Inclusion Criteria:

- Patients who are clinically diagnosed with homocystinuria

- Male/female patients aged 5 to 65 years

- Patients who consented and/or assented

Exclusion Criteria:

- Medically significant postnatal complications or congenital anomalies that are not
associated with homocystinuria

- Received any experimental therapy for homocystinuria during the 6 months prior to
enrollment or expected to receive any such therapy during duration of the study
We found this trial at
7
sites
Temple Street Children's University Hospital
1 Temple Street
Dublin, D01 XD99
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Children's Hospital of Philadelphia
South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Philadelphia, PA
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Children's Hospital Colorado
13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Aurora, CO
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Boston Children's Hospital
300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Boston, MA
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Emory University, School of Medicine Department of Human Genetics
2165 North Decatur Road
Decatur, Georgia 30033
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Decatur, GA
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Indiana University School of Medicine
340 W 10th St #6200
Indianapolis, Indiana 46202
(317) 274-3772
Indiana University School of Medicine With more than 2,000 students in 2013, the Indiana University...
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Indianapolis, IN
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The Children's National Medical Center (Institution)
Washington, District of Columbia 20010
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Washington,
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