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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07219485 | A Study of Pitolisant in Participants With Prader-Willi Syndrome | PHASE3 | ENROLLING BY_INVITATION | 150 | — | — | Aug 20, 2025 | Aug 1, 2030 | Mar 17, 2026 | 1 | United States |
| NCT06366464 | A Study of Pitolisant in Patients With Prader-Willi Syndrome | PHASE3 | RECRUITING | 134 | — | — | May 28, 2024 | Jul 1, 2027 | Mar 17, 2026 | 54 | United States, Australia +11 |
| NCT04257929 | A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension | PHASE2 | COMPLETED | 65 | — | — | Dec 9, 2020 | Jan 21, 2026 | May 12, 2026 | 13 | United States |
A treatment-emergent adverse event (TEAE) is any adverse event reported during treatment with study drug in this study and up to 30 days after final dose of study drug, or any worsening of a pre-existing condition reported during treatment with study drug and up to 30 days after final dose of study drug.
The PROMIS-SRI item bank consists of 13 items with a 5-point rating scale.
Change in Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) (parent/caregiver version) score from Baseline to Week 11 for pitolisant compared with placebo. The score of the ESS-CHAD ranges from 0 to 24. A decrease in score represents an improvement in excessive daytime sleepiness.
| Arm | Type | Description |
|---|---|---|
| Pitolisant | EXPERIMENTAL | All participants receive pitolisant administered orally once daily in the morning upon awakening. |
| Double-Blind Treatment Period Pitolisant | EXPERIMENTAL | Pitolisant tablets administered once daily in the morning upon wakening |
| Double-Blind Treatment Period Placebo | PLACEBO_COMPARATOR | Matching placebo administered tablets once daily in the morning upon wakening |
| Open-Label Extension Period Pitolisant | OTHER | Pitolisant tablets administered once daily in the morning upon wakening |
| Double-Blind Treatment Phase Lower Dose Pitolisant | ACTIVE_COMPARATOR | Pediatric patients (6 to less than 12 years of age): Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 8.9 mg pitolisant administered once daily in the morning. Adolescent patients (12 to less than 18 years of age): Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 13.35 mg pitolisant administered once daily in the morning. Adult patients (18 to 65 years of age): Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 17.8 mg pitolisant administered once daily in the morning. |
| Double-Blind Treatment Phase Higher Dose Pitolisant | ACTIVE_COMPARATOR | Pediatric patients (6 to less than 12 years of age): Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 17.8 mg pitolisant administered once daily in the morning. Adolescent patients (12 to less than 18 years of age): Week 1: 8.9 mg pitolisant administered once daily in the morning; Week 2: 17.8 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 26.7 mg pitolisant administered once daily in the morning. Adult patients (18 to 65 years of age): Week 1: 8.9 mg pitolisant administered once daily in the morning; Week 2: 17.8 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 35.6 mg pitolisant administered once daily in the morning. |
| Double-Blind Treatment Phase Placebo | PLACEBO_COMPARATOR | Pediatric patients (6 to less than 12 years of age): Week 1: Matching placebo tablets; Week 2: Matching placebo tablets; Weeks 3 through 11: Matching placebo tablets Adolescent patients (12 to less than 18 years of age): Week 1: Matching placebo tablets; Week 2: Matching placebo tablets; Weeks 3 through 11: Matching placebo tablets Adult patients (18 to 65 years of age): Week 1: Matching placebo tablets; Week 2: Matching placebo tablets; Weeks 3 through 11: Matching placebo tablets |
| Open-Label Pitolisant | OTHER | Age-based dosing (prior to implementation of amendment 6) or weight-based dosing (after implementation of amendment 6) |
| Name | Type | Description |
|---|---|---|
| Pitolisant | DRUG | * Pitolisant 4.45 mg tablets * Pitolisant 17.8 mg tablets |
| Pitolisant tablet | DRUG | Pitolisant tablet |
| Placebo tablet | OTHER | Placebo tablet |
| Pitolisant oral tablets | DRUG | Pitolisant 4.45 mg or 17.8 mg tablets |
| Placebo oral tablet | DRUG | Matching placebo tablets |
Inclusion Criteria: Each participant must meet the following criteria to be enrolled in this study: 1. Ability to provide voluntary, written informed consent (participant, if applicable, or parent\[s\]/legal guardian\[s\]) and, where applicable, voluntary, written assent (participant, as appropria...