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

Testing DNL952 for safety in adults with Late-Onset Pompe Disease.

Recruiting
18-75 years
All
Phase 1
**Study Overview:** This research is to check the safety of a new drug, **DNL952**, in adults with **Late-Onset Pompe Disease (LOPD)**, a condition that weakens muscles. The study is in **Phase 1**, meaning it's an early test to see if the drug is safe. We'll also study how the drug moves in the body (**pharmacokinetics**) and its effects (**pharmacodynamics**). **Who Can Join:** Participants must weigh at least 40 kg (about 88 pounds) and have LOPD. They should be able to walk 40 meters (about 130 feet) even with help. People in **Cohorts A1-A4** should have been on certain treatments like avalglucosidase alfa for a year. **Cohorts B1-B2** must not have had enzyme-replacement therapy for a year. **Who Cannot Join:** People with serious health issues not related to LOPD, those dependent on a wheelchair, or needing more than 6 hours of assisted breathing while awake, cannot participate. **Key Points:** - **Duration:** Participation involves multiple visits over time. - **Compensation:** Participants may receive compensation for their time. - **Risks:** As with any study, there may be risks or side effects, which will be monitored closely. Please consult your doctor to see if joining this study is suitable for you.
Study details
    Late-onset Pompe Disease

NCT07354724

Denali Therapeutics Inc.

10 July 2026

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