12 Pompe Disease Clinical Trials in Durham, NC (2026)

Q: Are there pompe disease clinical trials in Durham?

Yes, there are 12 pompe disease clinical trials currently recruiting in Durham, NC.

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

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

Q: Are clinical trials in Durham free?

Yes, clinical trial participation is always free. Study-related treatments, medical tests, and doctor visits are provided at no cost.

Recruiting Pompe Disease Studies in Durham

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...

Durham, NC500 participantsAmicus Therapeutics

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NCT04454216Recruiting

GSD VI and GSD IX Natural History

Collection and review of clinical information related to Glycogen Storage Disease Type VI (GSD VI) OR Glycogen Storage Disease Type IX (GSD IX) generated during clinic visits....

Durham, NC400 participantsDuke University

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NCT01665326Recruiting

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the diagnosis of Pompe disease, response to e...

Durham, NC400 participantsDuke University

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NCT02683512Recruiting

GBE Deficiency (GSD IV and APBD) Natural History Study

Collection and review of clinical information related to glycogen branching enzyme (GBE) deficiency, diagnosed as Glycogen Storage Disease Type IV (GSD IV) or Adult Polyglucosan Body Disease (APBD gen...

Durham, NC200 participantsDuke University

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NCT06795152Recruiting

Rare Glycogen Storage Diseases Natural History Study

The purpose of this study is to collect and study key medical data about several ultra-rare GSDs (Glycogen Storage Diseases) including, but not limited to, GSD types 0a, 0b, VII, X, XII, XIII, XV, PRK...

Durham, NC200 participantsDuke University

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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....

Durham, NC140 participantsUltragenyx Pharmaceutical Inc

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NCT04639336Recruiting

Cognitive and Neurological Pathologies in Pompe Disease

The purpose of this study is to better understand the long-term health effects of Pompe disease and to determine if there are any abnormal changes in the brain and peripheral nerves. Additionally, the...

Durham, NC45 participantsDuke University

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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....

Durham, NC45 participantsShionogi

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NCT04808505Recruiting

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric sub...

Durham, NC36 participantsAmicus Therapeutics

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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...

Durham, NC32 participantsDenali Therapeutics Inc.

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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...

Durham, NC20 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...

Durham, NC12 participantsAskBio Inc

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About Pompe Disease Clinical Trials in Durham

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 12 pompe disease clinical trials recruiting participants in Durham, NC. These studies are seeking a combined 2,030 participants. Research is being sponsored by Amicus Therapeutics, Duke University, Ultragenyx Pharmaceutical Inc and 4 other organizations. Clinical trial participation is free and participants receive study-related medical care at no cost.

Pompe Disease Clinical Trials in Durham — FAQ

Are there pompe disease clinical trials in Durham?

Yes, there are 12 pompe disease clinical trials currently recruiting in Durham, NC. Browse the studies on this page to find one that fits.

How do I join a clinical trial in Durham?

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

Are clinical trials in Durham free?

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

What pompe disease treatments are being tested?

The 12 active trials in Durham are testing new therapies including novel drugs, biologics, and treatment approaches for pompe disease.

Data updated July 13, 2026 from ClinicalTrials.gov