A Phase 1b/2a, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants with Late-Onset Pompe Disease

Status

Not yet recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2025-524082-25-00

Enrollment

Registered

2026-06-01

Start date

Unknown

Completion date

Unknown

Last updated

2026-06-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Late-Onset Pompe Disease

Brief summary

Incidence, severity, and seriousness of TEAEs ; Incidence and severity of IRRs

Detailed description

DNL952 serum PK parameters (when feasible): – Cmax – tmax – AUClast – AUC∞ (single dose only) – AUCt (multiple doses only) – t1/2, Incidence of ADAs during the study

Interventions

DRUGDNL952

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Incidence, severity, and seriousness of TEAEs ; Incidence and severity of IRRs	—

Secondary

Measure	Time frame
DNL952 serum PK parameters (when feasible): – Cmax – tmax – AUClast – AUC∞ (single dose only) – AUCt (multiple doses only) – t1/2, Incidence of ADAs during the study	—

Outcome results

None listed

Source: EU CTIS · Data processed: Jun 2, 2026