Pompe Disease (Late-onset) Clinical Trial
Official title:
Screening for Eligibility to Participate in a Clinical Trial of Gene Therapy for Late-onset Pompe Disease
NCT number | NCT03285126 |
Other study ID # | Pro00085445 |
Secondary ID | |
Status | Completed |
Phase | |
First received | |
Last updated | |
Start date | November 1, 2017 |
Est. completion date | June 29, 2019 |
Verified date | June 2019 |
Source | Duke University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
Status | Completed |
Enrollment | 19 |
Est. completion date | June 29, 2019 |
Est. primary completion date | June 29, 2019 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility |
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. |
Country | Name | City | State |
---|---|---|---|
United States | Duke University Medical Center | Durham | North Carolina |
Lead Sponsor | Collaborator |
---|---|
Duke University |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Eligibility for gene therapy. | The purpose of this study is to determine eligibility for the proposed clinical trial of gene therapy in adults with late-onset Pompe disease. Eligibility is measure by a composite score of blood tests, Electrocardiogram, Pulmonary Function Tests, and Muscle Status Testing. | Up to 2 days |
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