Recruiting
Phase 1

DNL952

Sponsor:

Denali Therapeutics Inc.

Code:

NCT07354724

Conditions

Late-onset Pompe Disease

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

DNL952

Study Details

Brief 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).

Conditions

Late-onset Pompe Disease

Study ID

NCT07354724

Start date

May 12, 2026

Status verified date

Aug, 2026

Completion date

Aug, 2028

Anticipated

Primary completion date

Aug, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

32 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort A1

Participants with LOPD

experimental: Cohort A2

Participants with LOPD

experimental: Cohort A3 (Optional)

Participants with LOPD

experimental: Cohort A4 (Optional)

Participants with LOPD

experimental: Cohort B1 (Optional)

Participants with LOPD

experimental: Cohort B2 (Optional)

Participants with LOPD

Interventions

DNL952

Intravenous repeating dose

Primary outcome measure

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

Central Contacts and Locations

Locations

University of California-Irvine

Recruiting

Irvine, California, United States, 92697

Contacts

Study Coordinator

714-790-8554kbjazevi@hs.uci.edu

Washington University School of Medicine

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Study Coordinator

314-362-6983koniak@wustl.edu

Duke University School of Medicine - Early Phase Research Unit

Recruiting

Durham, North Carolina, United States, 27710

Contacts

Study Coordinator

919-414-6998Janet.blount@duke.edu

The Lysosomal & Rare Disorders Research & Treatment Center

Recruiting

Fairfax, Virginia, United States, 22030

Contacts

Study Coordinator

571-732-4575aagha@ldrtc.org

More Information

Sponsor

Denali Therapeutics Inc.

Last update posted

Aug 17, 2026

Last verified

Aug, 2026

Keywords

  • LOPD
  • Pompe
  • acid maltase deficiency
  • glycogen storage disease type II
  • ERT
  • enzyme replacement therapy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Denali Therapeutics Inc. on 2026-08-17.