3 Pompe Disease Clinical Trials in Orange, CA (2026)

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

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

Recruiting Pompe Disease Studies in Orange

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...

Orange, CA2,000 participantsGenzyme, a Sanofi Company

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NCT06636383Recruiting

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration....

Orange, CA140 participantsUltragenyx Pharmaceutical Inc

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NCT06735755Recruiting

A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)

This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygou...

Orange, CA36 participantsBeam Therapeutics Inc.

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About Pompe Disease Clinical Trials in Orange

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 Orange, CA. These studies are seeking a combined 2,176 participants. Research is being sponsored by Genzyme, a Sanofi Company, Ultragenyx Pharmaceutical Inc, Beam Therapeutics Inc.. Clinical trial participation is free and participants receive study-related medical care at no cost.

Pompe Disease Clinical Trials in Orange — FAQ

Are there pompe disease clinical trials in Orange?

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

How do I join a clinical trial in Orange?

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

Are clinical trials in Orange free?

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

What pompe disease treatments are being tested?

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

Data updated July 13, 2026 from ClinicalTrials.gov