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).
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)
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
Sequential
DNL952
DrugIntravenous repeating dose
6
Treatment groupsSee each treatment group below.
Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs)
Incidence and severity of infusion-related reacations (IRRs)
PK parameter: Maximum concentration (Cmax) of DNL952 in serum
PK Parameter: Time to reach maximum concentration (tmax) of DNL952 in serum
PK Parameter: Area under the concentration-time curve (AUC) from time zero to time of last measurable concentration (AUClast) of DNL952 in serum
PK Parameter: AUC from time 0 to infinity (AUC∞) of DNL952 in serum
single dose only
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