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

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

Status

Recruiting

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07354724

Enrollment

Registered

2026-01-21

Start date

2026-05-12

Completion date

2028-08-01

Last updated

2026-08-17

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

Conditions

Late-onset Pompe Disease

Keywords

LOPD, Pompe, acid maltase deficiency, glycogen storage disease type II, ERT, enzyme replacement therapy

Brief summary

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

DRUGDNL952

Intravenous repeating dose

Study design

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 75 Years

Healthy volunteers

Inclusion criteria

Key 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

Design outcomes

Primary

Measure	Time frame
Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs)	48 weeks
Incidence and severity of infusion-related reacations (IRRs)	48 weeks

Secondary

Measure	Time frame	Description
PK parameter: Maximum concentration (Cmax) of DNL952 in serum	48 weeks	—
PK Parameter: Time to reach maximum concentration (tmax) of DNL952 in serum	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	48 weeks	—
PK Parameter: AUC from time 0 to infinity (AUC∞) of DNL952 in serum	48 weeks	single dose only
PK parameter: AUC from time zero to time t (AUCt) of DNL952 in serum	48 weeks	multiple doses only
PK Parameter: terminal elimination half-life (t1/2) of DNL952 in serum	48 weeks	—

Countries

United States

Contacts

STUDY_DIRECTORMedical Monitor

Denali Therapeutics Inc.

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Aug 18, 2026