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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07266324 | A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome. | PHASE2 | NOT YET_RECRUITING | 16 | — | — | Jan 1, 2026 | Mar 1, 2027 | Dec 5, 2025 | 2 | Australia |
The Hyperphagia Questionnaire for Clinical Trials consists of 9 items; each rated on a scale from 0 (no symptoms) to 4 (severe symptoms). The total score ranges from 0 to 36, with higher scores indicating worse hyperphagia symptoms. A score of approximately 13 is associated with moderate to severe hyperphagia, and a score of 22 or greater is associated with severe hyperphagia.
| Arm | Type | Description |
|---|---|---|
| BMB-101 | EXPERIMENTAL | Participants receive BMB-101 (10mg/mL liquid) orally |
| Placebo | PLACEBO_COMPARATOR | Participants receiving matched placebo orally |
| Name | Type | Description |
|---|---|---|
| BMB-101 | DRUG | Participants will receive weekly ascending oral doses of BMB-101(10 mg/mL) twice daily (BID) for 16 weeks. Doses will be based on weight (kg) and will initially start at 1.67 mg/kg. Doses may be titrated in 0.33 mg/kg increments based on tolerability up to a maximum dose of 2.0 mg/kg. |
| Placebo | DRUG | Matched Placebo |
Inclusion Criteria: * Participant must be aged 18-65 years (both inclusive). * Genetically confirmed diagnosis of Prader Willi Syndrome via standard DNA testing or other commonly approved methods. * Willing and able to provide voluntary written informed consent, or have a Legally Authorized Represe...