New drug DNL952 enters human testing for pompe disease
NCT ID NCT07354724
First seen Jun 27, 2026 · Last updated Jun 30, 2026 · Updated 2 times
Summary
This early-stage trial is testing a new drug called DNL952 in 32 adults with late-onset Pompe disease. The main goal is to check if the drug is safe and how the body processes it. Participants will receive the drug through an IV infusion at different doses.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- DNL952
- What this could lead to
- If successful, this could point toward a new treatment option for late-onset Pompe disease, potentially improving muscle function and quality of life.
- What could go wrong
- This is an early Phase 1 trial with only 32 participants, focused on safety. It is too small and early to know if DNL952 will be effective or have serious side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Duke University School of Medicine - Early Phase Research Unit
RECRUITINGDurham, North Carolina, 27710, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Lysosomal & Rare Disorders Research & Treatment Center
RECRUITINGFairfax, Virginia, 22030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of California-Irvine
RECRUITINGIrvine, California, 92697, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Major study aims to better measure muscular dystrophy progression
- Pompe disease patients invited to join worldwide registry
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