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Clinical Trials in the USA / NCT07354724
Recruiting Phase 1

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

NCT07354724 · tracked via the Priya Life Science USA tracker
Sponsor
Denali Therapeutics Inc.
Phase
Phase 1
Started
2026-05-12
Last updated
2026-08-17

Condition(s) studied

Late-onset Pompe Disease

Investigational drug(s) / intervention(s)

DNL952

DNL952: Intravenous repeating dose

Study 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

Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (4)

FacilityCityRegionStatus
University of California-Irvine Irvine California Recruiting
Washington University School of Medicine St Louis Missouri Recruiting
Duke University School of Medicine - Early Phase Research Unit Durham North Carolina Recruiting
The Lysosomal & Rare Disorders Research & Treatment Center Fairfax Virginia Recruiting

More Denali Therapeutics Inc. trials in the USA

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07354724 on ClinicalTrials.gov ↗ ← All trials in the USA