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Pompe Disease (Late-onset) Clinical Trials

Reviewed by TrialFinderData Editorial Team · Updated

4 recruiting trials for Pompe Disease (Late-onset). Eligibility criteria explained in plain English.

TrialFinderData lists 4 Pompe Disease (Late-onset) clinical trials drawn from ClinicalTrials.gov, all of which are currently recruiting participants.

Every phased trial in this set is Phase 1 / Phase 2 (2 of the listed studies); the remaining records are observational or have no phase recorded on ClinicalTrials.gov.

Research is led by University of Missouri-Columbia (1), GeneCradle Inc (1), Genzyme, a Sanofi Company (1), among the most active sponsors registered for these trials.

The most frequently studied intervention is Whole-body Electrical Muscle Stimulation Exercise (device, 1 trial), followed by GC301, AB-1009 (GAA Gene).

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.
4
Total Trials
4
Recruiting Now
0
Phase 3 Trials
4
Sponsors

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Recruiting Trials

Clinical trial data sourced from the ClinicalTrials.gov registry, maintained by the National Library of Medicine. Always consult your doctor before considering any clinical trial.

RECRUITINGNCT07478172

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults with neuromuscular...

Sponsor: University of Missouri-ColumbiaEnrolling: 501 location
RECRUITINGPhase 1 / Phase 2NCT06391736

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

This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as...

Sponsor: GeneCradle IncEnrolling: 331 location
RECRUITINGNCT00567073

Pompe Pregnancy Sub-Registry

This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the...

Sponsor: Genzyme, a Sanofi CompanyEnrolling: 2020 locations
RECRUITINGPhase 1 / Phase 2NCT07282847

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With...

This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with...

Sponsor: AskBio IncEnrolling: 129 locations

Frequently Asked Questions

There are currently 4 clinical trials for Pompe Disease (Late-onset), with 4 actively recruiting participants. These include trials across all phases from early-stage Phase 1 to late-stage Phase 3.

To join a clinical trial for Pompe Disease (Late-onset), review the eligibility criteria on the trial detail pages, then talk to your doctor about whether a trial is right for you. Your doctor can help you evaluate the potential benefits and risks.

Phase 3 trials are large-scale studies that test whether a treatment is effective and monitor side effects. There are 0 Phase 3 trials for Pompe Disease (Late-onset), representing treatments closest to potential FDA approval.

Clinical trials follow strict safety protocols overseen by Institutional Review Boards (IRBs) and the FDA. Participants are monitored closely and can withdraw at any time. Always discuss risks and benefits with your healthcare provider before enrolling.

Sources: ClinicalTrials.gov, FDA
Last updated:

Trial data sourced from the ClinicalTrials.gov API. This site does not provide medical advice, always talk to your doctor about clinical trial participation.