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

Conditions

• Late-onset Pompe Disease

Eligibility Criteria

Sex

ALL

Age

18 Years - 75 Years

Healthy Volunteers

Not accepted

Interventions

• DNL952 — DRUG
  Intravenous repeating dose

Study Details

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).

Key Dates

Start date

May 31, 2026

Status verified

May 2026

Primary completion

Aug 31, 2028

Completion

Aug 31, 2028

Study Design

Enrollment

32 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

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

Primary Outcome Measure

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

Locations (1)

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