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Clinical Trial Details — Status: Completed

Administrative data

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

Study information

Verified date June 2019
Source Duke University
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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.


Recruitment information / eligibility

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.

Study Design


Locations

Country Name City State
United States Duke University Medical Center Durham North Carolina

Sponsors (1)

Lead Sponsor Collaborator
Duke University

Country where clinical trial is conducted

United States, 

Outcome

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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Completed NCT03729362 - A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Phase 3
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