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DNL952 for Late-Onset Pompe Disease

This study is testing a drug called DNL952 in adults with late-onset Pompe disease (LOPD). The main goal is to understand if DNL952 is safe and how well your body tolerates it at different doses. Researchers will also look at how the drug moves through your body and what effects it has. You may be able to join if you are 18 to 75 years old, weigh at least 40 kg, have LOPD, and can walk at least 40 meters. Some participants will have received other Pompe disease treatments, while others will not. The study will measure side effects and reactions to infusions over 48 weeks. The current recruitment status is unclear.

Study design
This is a Phase 1, open-label study, meaning everyone knows what treatment is being given. It plans to enroll 32 adult participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will track side effects and infusion reactions for 48 weeks.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07354724

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

Recruiting
PHASE1Ages 18–75InterventionalTreatment
Denali Therapeutics Inc.
~32 participants
Updated 2026-08-17 on ClinicalTrials.gov
What's tested:DNL952

At a glance

Recruiting sites
4 of 4 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs)
Measured over 48 weeks
+1 more outcome measured

Conditions

Late-onset Pompe Disease

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Where you'd take part

This study runs at 4 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Duke University School of Medicine - Early Phase Research Unit

    Durham, North Carolinastudy coordinator listed

    Recruiting

  • The Lysosomal & Rare Disorders Research & Treatment Center

    Fairfax, Virginiastudy coordinator listed

    Recruiting

  • University of California-Irvine

    Irvine, Californiastudy coordinator listed

    Recruiting

  • Washington University School of Medicine

    St Louis, Missouristudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Medical Monitor · STUDY_DIRECTOR · Denali Therapeutics Inc.

Who to contact

Study Coordinator
[email protected]
714-790-8554
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

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Eligibility criteria

Inclusion

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

Exclusion

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

What this trial measures

  • Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs)48 weeks
  • Incidence and severity of infusion-related reacations (IRRs)48 weeks