A Phase 1 interventional study of DNL952 in Late-onset Pompe Disease, sponsored by Denali Therapeutics Inc.. Recruiting at 4 sites in United States. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-08-17.
Sponsored by Denali Therapeutics Inc. · Phase 1, Interventional, and Treatment
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).
162 studies on the registry are indexed under Glycogen Storage Disease Type II; 34 are open to participants now.
This study's planned enrollment of 32 is above the median of 20 across 89 interventional studies indexed under Glycogen Storage Disease Type II.
Browse Glycogen Storage Disease Type II studies →Denali Therapeutics Inc. is the lead sponsor of 19 studies on the registry; 5 are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 0 (0%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants with LOPD
Drug: DNL952
Participants with LOPD
Drug: DNL952
Participants with LOPD
Drug: DNL952
Participants with LOPD
Drug: DNL952
Participants with LOPD
Drug: DNL952
Participants with LOPD
Drug: DNL952
Intravenous repeating dose
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
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
single dose only
Time frame: 48 weeks
PK parameter: AUC from time zero to time t (AUCt) of DNL952 in serum
multiple doses only
Time frame: 48 weeks
PK Parameter: terminal elimination half-life (t1/2) of DNL952 in serum
Time frame: 48 weeks
Plan to share: No
No publications or documents are linked to this record.
Started May 2026 (actual). Primary completion is estimated for Aug 2028, 27 months after the start.
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No contact was published for this record. The registry link below has the sponsor’s details.
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Glycogen Storage Disease Type II →
Denali Therapeutics Inc.