Pompe Gene Therapy- Screening for Eligibility

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

19 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-11-01

Study Completion Date

2019-06-29

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

NCT06391736

Pompe Disease (Late-onset)

RECRUITING PHASE1/PHASE2

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

NCT06150820

Pompe Disease (Late-onset)

ACTIVE_NOT_RECRUITING NA

A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease

NCT00515398

Pompe Disease Glycogen Storage Disease Type II

COMPLETED

Gene Transfer Study in Patients With Late Onset Pompe Disease

NCT04174105

Pompe Disease (Late-onset)

ACTIVE_NOT_RECRUITING PHASE1/PHASE2

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

NCT01230801

Pompe Disease

COMPLETED PHASE1/PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Pompe Disease (Late-onset)

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No