ClinicalTrials.gov record NCT07354724

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

A Phase 1, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 1An early-stage study, usually small, mainly to check safety, side effects and how much of a treatment can be given. Whether it works is not the main question yet.
Age18 years old to 75 years old
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Phase 1, open-label study in adults with late-onset Pompe disease. It evaluates safety and tolerability of repeated intravenous doses of DNL952 across several dose levels and measures how the drug behaves in the body and its biological effects (PK/PD). Participants are placed into sequential, non-randomized cohorts and monitored for adverse events and infusion-related reactions.

What it is studying. The study is collecting safety and tolerability data for DNL952 at various dose levels in adults with late-onset Pompe disease. It also measures pharmacokinetics (drug levels over time) and pharmacodynamics (the drug's biological effects) and records infusion-related reactions.

What the researchers hope to learn. Obtain safety and tolerability data for DNL952 in adults with late-onset Pompe disease; characterize pharmacokinetic and pharmacodynamic profiles; document incidence and severity of treatment-emergent adverse events and infusion-related reactions.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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).

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • DNL952

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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 description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will receive repeated intravenous (IV) doses of DNL952. They will have blood samples taken to measure how the drug behaves (pharmacokinetics) and its effects (pharmacodynamics), and will be closely monitored for side effects including infusion-related reactions. Study visits, monitoring, and measurements continue through about 48 weeks for the safety and PK/PD outcomes reported in the registry.

How the study is structured. A Phase 1 interventional trial that is open-label (no masking) and non-randomized, using sequential cohorts. Adults receive repeated intravenous doses of DNL952 and are followed for safety, infusion reactions, and pharmacokinetic/pharmacodynamic measurements, with primary assessments reported through 48 weeks.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Pompe disease

Clinical trials32 trials listed for Pompe diseaseEvery trial the registry lists for Pompe disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Pompe disease researchLatest monthly update: September 2026. Includes: Antibody effects. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
May 12, 2026
Primary completion
August 2028 (estimated)
Study completion
August 2028 (estimated)
Target enrollment
32 participants (estimated)
Last updated on registry
Aug 17, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Denali Therapeutics Inc.

Participating sites (4)

  • University of California-IrvineRecruiting

    Irvine, California, 92697 · United States

  • Washington University School of MedicineRecruiting

    St Louis, Missouri, 63110 · United States

  • Duke University School of Medicine - Early Phase Research UnitRecruiting

    Durham, North Carolina, 27710 · United States

  • The Lysosomal & Rare Disorders Research & Treatment CenterRecruiting

    Fairfax, Virginia, 22030 · United States

Terms used on this page · explanations by MedicalUpdates.ai

open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
pharmacokinetics
How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
pharmacodynamics
What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.
FVC
A breathing test that measures the total amount of air a person can blow out after a full breath in. It is often given as a percentage of what is expected for a person's age and size.
prior to screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Sponsor
The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
investigational drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
5 half-lives
The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
Key Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Key Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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