Pompe Gene Therapy- Screening for Eligibility

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03285126
• Other study ID #: Pro00085445
• Status: Completed
• 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
• 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.

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Pompe Disease (Late-onset)

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