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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07304843 | A Two-part Study to Investigate the Effects in Adults of Two Doses of Golexanolone in Patients With Primary Biliary Cholangitis (PBC) With Fatigue and Cognitive Dysfunction | PHASE1 | RECRUITING | 84 | — | — | Apr 14, 2023 | Jun 30, 2026 | Mar 9, 2026 | 36 | Germany, Greece +6 |
Frequency, intensity, and seriousness of adverse events (AEs) from baseline to Day 28
Frequency, intensity, and seriousness of adverse events (AEs)
| Arm | Type | Description |
|---|---|---|
| Part B: Treatment arm 1 (golexanolone 40 mg) | EXPERIMENTAL | 40 mg golexanolone BID |
| Part B: Treatment arm 2 (golexanolone 80 mg) | EXPERIMENTAL | 80 mg golexanolone BID |
| Placebo | PLACEBO_COMPARATOR | Part B: Placebo BID |
| Part A: Golexanolone | EXPERIMENTAL | 40 mg golexanolone BID |
| Part A: Placebo | PLACEBO_COMPARATOR | Placebo BID |
| Name | Type | Description |
|---|---|---|
| golexanolone | DRUG | soft gelatin capsules, oral dosage twice per day for up to 28 days |
| Placebo | DRUG | soft gelatin capsules, oral dosage twice a day for up to 28 days |
Inclusion Criteria: * Male and female subjects age ≥ 18 years * Diagnosis of PBC based on the presence of ≥2 of 3 key disease characteristics * Clinically significant fatigue defined for the purposes of this study as a PBC-40 fatigue domain score of ≥29 at screening * Clinically significant cogniti...