Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07354724 | A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease | PHASE1 | RECRUITING | 32 | — | — | May 1, 2026 | Aug 1, 2028 | May 12, 2026 | 1 | United States |
| Arm | Type | Description |
|---|---|---|
| Cohort A1 | EXPERIMENTAL | Participants with LOPD |
| Cohort A2 | EXPERIMENTAL | Participants with LOPD |
| Cohort A3 (Optional) | EXPERIMENTAL | Participants with LOPD |
| Cohort A4 (Optional) | EXPERIMENTAL | Participants with LOPD |
| Cohort B1 (Optional) | EXPERIMENTAL | Participants with LOPD |
| Cohort B2 (Optional) | EXPERIMENTAL | Participants with LOPD |
| Name | Type | Description |
|---|---|---|
| DNL952 | DRUG | Intravenous repeating dose |
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 we...