Clinical Trials Logo

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
See also
  Status Clinical Trial Phase
Completed NCT00158600 - A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease Phase 3
Recruiting NCT04174105 - Gene Transfer Study in Patients With Late Onset Pompe Disease Phase 1/Phase 2
Active, not recruiting NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Phase 1/Phase 2
Recruiting NCT05083806 - MSOT in Pompe Disease N/A
Recruiting NCT05272969 - Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease
Active, not recruiting NCT03694561 - Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
Completed NCT00731081 - Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme® N/A
Completed NCT00455195 - Late-Onset Treatment Study Extension Protocol Phase 4
Recruiting NCT05951790 - Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease N/A
Completed NCT01288027 - Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa Phase 4
Recruiting NCT00567073 - Pompe Pregnancy Sub-Registry
Completed NCT03893240 - Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease N/A
Withdrawn NCT04094948 - Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Phase 2
Completed NCT00250939 - A Study of rhGAA in Patients With Late-Onset Pompe Disease Phase 2
Recruiting NCT03911505 - ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD Phase 3
Recruiting NCT06150820 - A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease N/A
Completed NCT03729362 - A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Phase 3
Not yet recruiting NCT06178432 - Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD) Early Phase 1
Completed NCT00268944 - Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support Phase 3
Active, not recruiting NCT04138277 - A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD Phase 3