Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
DCCR · 3 trials · 1 indication
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...