2 Pompe Disease Clinical Trials in Fairfax, VA (2026)

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

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

Recruiting Pompe Disease Studies in Fairfax

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....

Fairfax, VA45 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...

Fairfax, VA32 participantsDenali Therapeutics Inc.

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About Pompe Disease Clinical Trials in Fairfax

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 2 pompe disease clinical trials recruiting participants in Fairfax, VA. These studies are seeking a combined 77 participants. Research is being sponsored by Shionogi, Denali Therapeutics Inc.. Clinical trial participation is free and participants receive study-related medical care at no cost.

Pompe Disease Clinical Trials in Fairfax — FAQ

Are there pompe disease clinical trials in Fairfax?

Yes, there are 2 pompe disease clinical trials currently recruiting in Fairfax, VA. Browse the studies on this page to find one that fits.

How do I join a clinical trial in Fairfax?

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

Are clinical trials in Fairfax free?

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

What pompe disease treatments are being tested?

The 2 active trials in Fairfax are testing new therapies including novel drugs, biologics, and treatment approaches for pompe disease.

Data updated July 13, 2026 from ClinicalTrials.gov