NCT07354724

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).

Trial Health

65
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
32

participants targeted

Target at P50-P75 for phase_1

Timeline
27mo left

Started Mar 2026

Typical duration for phase_1

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress8%
Mar 2026Aug 2028

First Submitted

Initial submission to the registry

January 12, 2026

Completed
9 days until next milestone

First Posted

Study publicly available on registry

January 21, 2026

Completed
1 month until next milestone

Study Start

First participant enrolled

March 1, 2026

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2028

Last Updated

January 21, 2026

Status Verified

January 1, 2026

Enrollment Period

2.4 years

First QC Date

January 12, 2026

Last Update Submit

January 12, 2026

Conditions

Late-onset Pompe Disease

Keywords

LOPDPompeacid maltase deficiencyglycogen storage disease type IIERTenzyme replacement therapy

Outcome Measures

Primary Outcomes (2)

  • Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs)

    48 weeks

  • Incidence and severity of infusion-related reacations (IRRs)

    48 weeks

Secondary Outcomes (6)

  • 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

  • PK parameter: AUC from time zero to time t (AUCt) of DNL952 in serum

    48 weeks

  • +1 more secondary outcomes

Study Arms (6)

Cohort A1

EXPERIMENTAL

Participants with LOPD

Drug: DNL952

Cohort A2

EXPERIMENTAL

Participants with LOPD

Drug: DNL952

Cohort A3 (Optional)

EXPERIMENTAL

Participants with LOPD

Drug: DNL952

Cohort A4 (Optional)

EXPERIMENTAL

Participants with LOPD

Drug: DNL952

Cohort B1 (Optional)

EXPERIMENTAL

Participants with LOPD

Drug: DNL952

Cohort B2 (Optional)

EXPERIMENTAL

Participants with LOPD

Drug: DNL952

Interventions

DNL952DRUG

Intravenous repeating dose

Cohort A1Cohort A2Cohort A3 (Optional)Cohort A4 (Optional)Cohort B1 (Optional)Cohort B2 (Optional)

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Medical Monitor

    Denali Therapeutics Inc.

    STUDY DIRECTOR

Central Study Contacts

Clinical Trials at Denali Therapeutics

CONTACT

Email:clinical-trials@dnli.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 12, 2026

First Posted

January 21, 2026

Study Start

March 1, 2026

Primary Completion (Estimated)

August 1, 2028

Study Completion (Estimated)

August 1, 2028

Last Updated

January 21, 2026

Record last verified: 2026-01

Data Sharing

IPD Sharing
Will not share