3 Pompe Disease Clinical Trials in New York, NY (2026)

Q: How do I join a clinical trial in New York?

Browse available studies, click one that interests you, and complete the free eligibility form. A study coordinator at the New York research site will contact you.

Recruiting Pompe Disease Studies in New York

NCT06121011Recruiting

A Global Prospective Observational Registry of Patients With Pompe Disease

This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both u...

New York, NY500 participantsAmicus Therapeutics

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NCT00567073Recruiting

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 Pompe Registry, regard...

New York, NY20 participantsGenzyme, a Sanofi Company

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NCT07282847Recruiting

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)

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 late-onset Pompe diseas...

New York, NYPHASE112 participantsAskBio Inc

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About Pompe Disease Clinical Trials in New York

Pompe disease is a rare genetic disorder caused by accumulation of glycogen in cells due to deficiency of the enzyme acid alpha-glucosidase. It can affect the heart, skeletal muscles, and respiratory system. Treatment includes enzyme replacement therapy.

There are currently 3 pompe disease clinical trials recruiting participants in New York, NY. These studies are seeking a combined 532 participants. Research is being sponsored by Amicus Therapeutics, Genzyme, a Sanofi Company, AskBio Inc. Clinical trial participation is free and participants receive study-related medical care at no cost.

Pompe Disease Clinical Trials in New York — FAQ

Are there pompe disease clinical trials in New York?

Yes, there are 3 pompe disease clinical trials currently recruiting in New York, NY. Browse the studies on this page to find one that fits.

How do I join a clinical trial in New York?

Browse the studies listed above, click one that interests you, and complete the free eligibility form. A study coordinator at the New York research site will contact you about next steps.

Are clinical trials in New York free?

Yes, clinical trial participation is always free. Study-related treatment, medical tests, and doctor visits are provided at no cost. Many New York studies also compensate for your time and travel.

What pompe disease treatments are being tested?

The 3 active trials in New York are testing new therapies including novel drugs, biologics, and treatment approaches for pompe disease.

Data updated September 1, 2026 from ClinicalTrials.gov