A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: DNL952.
- Who it may be relevant to
- Registry conditions: Late-onset Pompe Disease. Basic parameters: 18 years — 75 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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
- Drug DNL952
Intravenous repeating dose
Primary outcome measures
- 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]
Secondary outcome measures (6)
- PK parameter: Maximum concentration (Cmax) of DNL952 in serum [Time frame: 48 weeks]
- PK Parameter: Time to reach maximum concentration (tmax) of DNL952 in serum [Time frame: 48 weeks]
- PK Parameter: Area under the concentration-time curve (AUC) from time zero to time of last measurable concentration (AUClast) of DNL952 in serum [Time frame: 48 weeks]
- PK Parameter: AUC from time 0 to infinity (AUC∞) of DNL952 in serum [Time frame: 48 weeks]
- PK parameter: AUC from time zero to time t (AUCt) of DNL952 in serum [Time frame: 48 weeks]
- PK Parameter: terminal elimination half-life (t1/2) of DNL952 in serum [Time frame: 48 weeks]
Eligibility criteria
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
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 3 centers
- University of California-Irvine — Irvine
- Duke University School of Medicine - Early Phase Research Unit — Durham
- The Lysosomal & Rare Disorders Research & Treatment Center — Fairfax
Identifiers
NCT: NCT07354724 · DNLI-J-0001 · 2025-524082-25-00