Pompe Gene Therapy- Screening for Eligibility
| Status: | Recruiting |
|---|---|
| Conditions: | Diabetes |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 9/5/2018 |
| Start Date: | November 1, 2017 |
| End Date: | August 1, 2019 |
| Contact: | Stephanie DeArmey |
| Email: | stephanie.dearmey@duke.edu |
| Phone: | 9196811946 |
Screening for Eligibility to Participate in a Clinical Trial of Gene Therapy for Late-onset Pompe Disease
The purpose of this study is to determine eligibility for the future clinical trial of gene
therapy in adults with late-onset Pompe disease.
This screening protocol will enroll up to 20 adults patients (>=18 yo) with late-onset Pompe
disease. Study assessments include review of medical history, vital signs, physical
examination, muscle function testing, lung function testing, blood and urine collection,
serum pregnancy test and ECG.
The study results will be collected to perform descriptive statistical analysis and used to
determine eligibility for the proposed clinical trial of gene therapy in the future.
therapy in adults with late-onset Pompe disease.
This screening protocol will enroll up to 20 adults patients (>=18 yo) with late-onset Pompe
disease. Study assessments include review of medical history, vital signs, physical
examination, muscle function testing, lung function testing, blood and urine collection,
serum pregnancy test and ECG.
The study results will be collected to perform descriptive statistical analysis and used to
determine eligibility for the proposed clinical trial of gene therapy in the future.
Inclusion Criteria
- Diagnosis of Pompe disease by blood or skin fibroblast GAA assay and two pathogenic
variants in the GAA gene.
- Age: at least 18 years at enrollment.
- Subjects are capable of giving written consent.
- Receiving ERT at a stable dose for at least 104 weeks.
Exclusion Criteria
- Any condition that would interfere with participation in the study as determined by
the principal investigator.
- Pregnancy or nursing mothers.
- History of active hepatitis B, hepatitis C, or cirrhosis
- Receiving any investigational agent.
We found this trial at
1
site
2301 Erwin Rd
Durham, North Carolina 27710
Durham, North Carolina 27710
919-684-8111
Principal Investigator: Dwight Koeberl
Phone: 919-681-1946
Duke Univ Med Ctr As a world-class academic and health care system, Duke Medicine strives...
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