Late-onset Pompe DiseasePhase 1NCT07354724
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
A Phase 1 study testing DNL952 in people with Late-onset Pompe Disease, run by Denali Therapeutics Inc. at 4 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Late-onset Pompe Disease
- What is being tested?
- DNL952
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 4 sitesUnited States
- Study dates
- 12 May 2026to Aug 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Duke University School of Medicine - Early Phase Research Unit
Durham, North Carolina, 27710
Study Coordinator, 919-414-6998, Janet.blount@duke.edu
- Recruiting
The Lysosomal & Rare Disorders Research & Treatment Center
Fairfax, Virginia, 22030
Study Coordinator, 571-732-4575, aagha@ldrtc.org
- Recruiting
University of California-Irvine
Irvine, California, 92697
Study Coordinator, 714-790-8554, kbjazevi@hs.uci.edu
- Recruiting
Washington University School of Medicine
St Louis, Missouri, 63110
Study Coordinator, 314-362-6983, koniak@wustl.edu
About this study
Official title: 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
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).
- Conditions
- Late-onset Pompe Disease
- Being tested
- DNL952
- Keywords
- LOPD, Pompe, acid maltase deficiency, glycogen storage disease type II, ERT, enzyme replacement therapy
- Medical subject headings
- Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DNL952Drug
Intravenous repeating dose
| Study group | Role | What they receive |
|---|---|---|
| Cohort A1 | Experimental | Participants with LOPD Receives: Drug: DNL952 |
| Cohort A2 | Experimental | Participants with LOPD Receives: Drug: DNL952 |
| Cohort A3 (Optional) | Experimental | Participants with LOPD Receives: Drug: DNL952 |
| Cohort A4 (Optional) | Experimental | Participants with LOPD Receives: Drug: DNL952 |
| Cohort B1 (Optional) | Experimental | Participants with LOPD Receives: Drug: DNL952 |
| Cohort B2 (Optional) | Experimental | Participants with LOPD Receives: Drug: DNL952 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs)
Measured: 48 weeks
Incidence and severity of infusion-related reacations (IRRs)
Measured: 48 weeks
Secondary outcomes (6)
PK parameter: Maximum concentration (Cmax) of DNL952 in serum
Measured: 48 weeks
PK Parameter: Time to reach maximum concentration (tmax) of DNL952 in serum
Measured: 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
Measured: 48 weeks
PK Parameter: AUC from time 0 to infinity (AUC∞) of DNL952 in serum
single dose only
Measured: 48 weeks
PK parameter: AUC from time zero to time t (AUCt) of DNL952 in serum
multiple doses only
Measured: 48 weeks
PK Parameter: terminal elimination half-life (t1/2) of DNL952 in serum
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jan 2026
- Study start12 May 2026
- Primary completionAug 2028 (estimated)
- Study completionAug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Late-onset Pompe DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Denali Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Denali Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07354724
- Sponsor's protocol ID
- DNLI-J-0001
- Other IDs
- 2025-524082-25-00, 2025
- Status last verified
- Aug 2026
- First submitted
- 12 Jan 2026
- Last updated
- 17 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07354724
- Acronym / sponsor ID
- DNLI-J-0001
- Conditions
- Late-onset Pompe Disease
- Keywords
- LOPD, Pompe, acid maltase deficiency, glycogen storage disease type II, ERT, enzyme replacement therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Denali Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 12 May 2026 (actual)
- Primary completion
- Aug 2028 (estimated)
- Study completion
- Aug 2028 (estimated)
- First posted
- 21 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Cohort A1 | Experimental | Drug: DNL952Participants with LOPD |
| Cohort A2 | Experimental | Drug: DNL952Participants with LOPD |
| Cohort A3 (Optional) | Experimental | Drug: DNL952Participants with LOPD |
| Cohort A4 (Optional) | Experimental | Drug: DNL952Participants with LOPD |
| Cohort B1 (Optional) | Experimental | Drug: DNL952Participants with LOPD |
| Cohort B2 (Optional) | Experimental | Drug: DNL952Participants with LOPD |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DNL952Intravenous repeating dose | Drug | Cohort A1, Cohort A2, Cohort A3 (Optional), Cohort A4 (Optional), Cohort B1 (Optional), Cohort B2 (Optional) |
Primary outcome
Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs)
Time frame: 48 weeks
Incidence and severity of infusion-related reacations (IRRs)
Time frame: 48 weeks
6 secondary outcomes
PK parameter: Maximum concentration (Cmax) of DNL952 in serum
Time frame: 48 weeks
PK Parameter: Time to reach maximum concentration (tmax) of DNL952 in serum
Time frame: 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
Time frame: 48 weeks
PK Parameter: AUC from time 0 to infinity (AUC∞) of DNL952 in serum
Time frame: 48 weeks
PK parameter: AUC from time zero to time t (AUCt) of DNL952 in serum
Time frame: 48 weeks
PK Parameter: terminal elimination half-life (t1/2) of DNL952 in serum
Time frame: 48 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Duke University School of Medicine - Early Phase Research Unit
Durham, United States
Medical Monitor study director
Denali Therapeutics Inc.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing DNL952 in people with Late-onset Pompe Disease, run by Denali Therapeutics Inc. at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs) (time frame: 48 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Aug 2026.