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NCT07354724PHASE1Recruiting

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

Denali Therapeutics Inc.

Start Date

5/12/2026

Completion Date

8/1/2028

Summary

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. The principal aim of this study is to obtain safety and tolerability data across varous dose levels of DNL952 in participants with late-onset Pompe disease (LOPD).

Eligibility Criteria

Age Range: 18 years to 75 years

Key Inclusion Criteria: * Body weight ≥40 kg * Diagnosis of LOPD * Upright FVC ≥ 30% of predicted normal value * Able to ambulate ≥ 40 meters (use of assistive devices is acceptable) * \[Cohorts A1-A4 only\] Have received avalglucosidase alfa or cipaglucosidase alfa at a dose of 20 mg/kg every 2 weeks for at least 12 months prior to screening * \[Cohorts B1-B2 only\] Must not have received any enzyme-replacement therapy for Pompe disease in the 12 months prior to screening Key Exclusion Criteria: * Any ongoing, clinically significant, unstable, or poorly controlled neurological, psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematological, immunological, allergic, or ophthalmic disease not related to Pompe disease, or other major disorders. Well-controlled conditions are permitted if investigator and Sponsor agree. * Wheelchair-dependent * Require noninvasive ventilation for an average of more than 6 hours per day while awake or any invasive ventilation. Use of noninvasive ventilation during sleep is acceptable. * Received an experimental gene therapy at any time or participation in any other investigational drug trial or use of investigational drug within 60 days or 5 half-lives, whichever is longer, before screening

Interventions

DRUG

DNL952

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Conditions

Late-onset Pompe Disease

Locations

University of California-Irvine

Irvine, California 92697

United States

Duke University School of Medicine - Early Phase Research Unit

Durham, North Carolina 27710

United States

The Lysosomal & Rare Disorders Research & Treatment Center

Fairfax, Virginia 22030

United States