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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05643872 | A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita | PHASE3 | RECRUITING | 45 | — | — | Nov 15, 2022 | Nov 1, 2023 | Dec 9, 2022 | 1 | United States |
| NCT04520750 | VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita | PHASE3 | COMPLETED | 36 | — | — | Sep 1, 2020 | Dec 15, 2022 | Aug 29, 2024 | 4 | United States |
| NCT03920228 | Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita | PHASE2 | COMPLETED | 73 | — | — | Apr 1, 2019 | Oct 19, 2020 | Aug 29, 2024 | 9 | United States |
| Arm | Type | Description |
|---|---|---|
| PTX-022 | EXPERIMENTAL | - |
| Open label treatment arm | EXPERIMENTAL | PTX-022 QTORIN |
| Open label period | EXPERIMENTAL | - |
| Randomized period - Dosing A | PLACEBO_COMPARATOR | - |
| Randomized period - Dosing B | EXPERIMENTAL | - |
| Randomized period - Dosing C | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| PTX-022 | DRUG | QTORIN rapamycin 3.9% anhydrous gel |
| Placebo | DRUG | Placebo topical |
Key Inclusion Criteria: * Completed the PALV-05 (VAPAUS) study * Agree to contraceptive use Key Exclusion Criteria: * Females who are pregnant or breastfeeding * Concomitant use of sirolimus or any sirolimus-containing medications (systemic or topical) * Any significant concurrent condition that ...