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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05701774 | Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome | PHASE3 | ACTIVE NOT_RECRUITING | 83 | — | — | Jan 31, 2023 | Jun 1, 2028 | Apr 2, 2025 | 22 | United States, United Kingdom |
| NCT03714373 | Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period | PHASE3 | COMPLETED | 115 | — | — | Oct 1, 2018 | Aug 17, 2023 | Apr 19, 2024 | 28 | United States, United Kingdom |
| NCT03440814 | A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome | PHASE3 | COMPLETED | 127 | — | — | May 9, 2018 | May 1, 2020 | Sep 21, 2023 | 29 | United States, United Kingdom |
Assess the safety of DCCR by evaluating the incidence and severity of adverse events. Safety analyses will be conducted in all subjects who receive at least one dose of DCCR in the C614 study. Adverse events will be described by type and level of severity.
Safety analyses will be conducted in all participants who receive at least one dose of DCCR. Adverse events will be described by type and level of severity.
Hyperphagia-related behaviors will be assessed by the hyperphagia questionnaire for clinical trials (HQ-CT), an instrument designed to measure symptoms of food related preoccupations and behaviors. The HQ-CT consists of nine items with responses ranging from 0-4 (best to worst). Scores from 9 items will be summed for a possible total score range of 0-36.
Hyperphagia-related behaviors were assessed by the validated hyperphagia questionnaire for clinical trials (HQ-CT), an instrument designed to measure symptoms of food related preoccupations and behaviors that was completed by the caregiver. The HQ-CT consists of nine items with responses ranging from 0-4 units each (possible total score range: 0-36). The HQ-CT was assessed at Screening, Baseline (Visit 2), and approximately every 4 weeks post-dose at Week 4, Week 8, and Week 13. A decrease in score from baseline represented improvement.
| Arm | Type | Description |
|---|---|---|
| DCCR | EXPERIMENTAL | 75 - 525 mg DCCR |
| OLE DCCR | EXPERIMENTAL | 75 - 525 mg DCCR |
| RW DCCR | EXPERIMENTAL | 75 - 525 mg DCCR |
| RW Placebo | PLACEBO_COMPARATOR | 75 - 525 mg Placebo for DCCR |
| Placebo | PLACEBO_COMPARATOR | 75 - 450 mg placebo for DCCR |
| Name | Type | Description |
|---|---|---|
| DCCR | DRUG | Once daily oral administration |
| Placebo for DCCR | DRUG | Once daily oral administration of double-blind (placebo for DCCR) tablet(s) during the RW Period |
Inclusion Criteria: 1. Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (subjects, as appropriate) 2. Participant must: 1. Have participated in and completed the Study C602 Randomized Withdrawal (RW) Period; 2. Have par...