5 Pompe Disease Clinical Trials in Irvine, CA (2026)

Q: How do I join a clinical trial in Irvine?

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

Recruiting Pompe Disease Studies in Irvine

NCT00231400Recruiting

Pompe Disease Registry Protocol

The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outc...

Irvine, CA2,000 participantsGenzyme, a Sanofi Company

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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...

Irvine, CA500 participantsAmicus Therapeutics

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NCT07123155Recruiting

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT....

Irvine, CAPHASE245 participantsShionogi

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NCT07354724Recruiting

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

This is a Phase 1, multicenter, open-label study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL952 in adult participants with late-onset Pompe disease. T...

Irvine, CAPHASE132 participantsDenali Therapeutics Inc.

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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...

Irvine, CAPHASE112 participantsAskBio Inc

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About Pompe Disease Clinical Trials in Irvine

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 5 pompe disease clinical trials recruiting participants in Irvine, CA. These studies are seeking a combined 2,589 participants. Research is being sponsored by Genzyme, a Sanofi Company, Amicus Therapeutics, Shionogi and 2 other organizations. Clinical trial participation is free and participants receive study-related medical care at no cost.

Pompe Disease Clinical Trials in Irvine — FAQ

Are there pompe disease clinical trials in Irvine?

Yes, there are 5 pompe disease clinical trials currently recruiting in Irvine, CA. Browse the studies on this page to find one that fits.

How do I join a clinical trial in Irvine?

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

Are clinical trials in Irvine free?

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

What pompe disease treatments are being tested?

The 5 active trials in Irvine are testing new therapies including novel drugs, biologics, and treatment approaches for pompe disease.

Data updated October 16, 2026 from ClinicalTrials.gov