Treatment of Hyperphagia Behavioral Symptoms in Children and Adults Diagnosed With Prader-Willi Syndrome

The HPWSQ-R is an 11-item questionnaire examining the psychological, developmental, and neurobiological correlates of hyperphagia in PWS. The items are classified into 3 domains; behavior, drive, and severity with each item rated on a five-point scale (1: not at all/none of the time/extremely easy to 5: extremely/all of the time/extremely hard). The questionnaire was completed by the parent/caregiver using a 1-week recall period. Total score was the sum of all the items in the three domains and ranged from 11 (no hyperphagia behaviors) to 55 (most severe hyperphagia behaviors). Change from baseline in HPWSQ-R Total Score at Day 15 is presented for this outcome measure.

Time frame: From Day 1 (baseline) to Day 15

Population: The analysis population included the full analysis set (FAS) which consisted of all subjects in the safety population who had the primary efficacy endpoint measured (at baseline and phone call \[Day 8\] ±1 or Day 15). Analyses for the FAS were conducted according to the randomized treatment.

The CY-BOCS is a clinician rated, semi-structured inventory of specific symptoms and symptom severity in pediatric obsessive-compulsive disorder (OCD). Total scores on the CY-BOCS are calculated using a symptom checklist and severity scale. The 10 severity items are summed to produce an Obsessions Severity Score (5 items), Compulsions Severity Score (5 items), and Total score (sum of all 10 severity items). The total score is calculated by summing the 10 individual scores and ranges from 0 (no obsessions or compulsions) to 40 (most severe OC).

Time frame: From Day 1 (baseline) to Day 15

Population: The analysis population included the FAS which consisted of all subjects in the safety population who had the primary efficacy endpoint measured (at baseline and phone call \[Day 8\] ±1 or Day 15). Analyses for the FAS were conducted according to the randomized treatment.~Number of subjects analyzed signifies those subjects who were evaluable for this endpoint

The HPWSQ-R is an 11-item questionnaire examining the psychological, developmental, and neurobiological correlates of hyperphagia in PWS. The items were classified into 3 domains; behavior, drive, and severity with each item rated on a five-point score range from 1 (not at all/none of the time/extremely easy) to 5 (extremely/all of the time/extremely hard). The questionnaire was completed by the parent/caregiver using a 1-week recall period. Changes in the HPWSQ-R Total Score and in the Domain Scores (behavior, drive, and severity) at Day 15 is presented for this outcome measure. HPWSQ-R Behavior, Drive and Severity scores range from 5-25, 4-20, and 2-10, respectively, with higher scores indicating a worse outcome. Change from baseline is presented = (Day 15 score minus Baseline score).

Time frame: From Day 1 (baseline) to Day 15

Population: The analysis population included the FAS which consisted of all subjects in the safety population who had the primary efficacy endpoint measured (at baseline and phone call \[Day 8\] ±1 or Day 15). Analyses for the FAS were conducted according to the randomized treatment.

The Food Domain Score of the Reiss Profile consisted of 7 questions that pertain to food seeking behavior. The questions were rated on a five point scale ranging from -2 (strongly disagree; this phrase is not at all characteristic of the person) to 2 (strongly agree; this phrase is definitely characteristic of the person). Total score was defined as the sum of all individual item scores. The total score ranged from -14 to 14 with higher scores indicating higher severity. Change from baseline in Food Domain of the Reiss Profile at Day 15 is presented for this outcome measure.

Time frame: From Day 1 (baseline) to Day 15

Population: The analysis population included the FAS which consisted of all subjects in the safety population who had the primary efficacy endpoint measured (at baseline and phone call \[Day 8\] ±1 or Day 15). Analyses for the FAS were conducted according to the randomized treatment.~Number of subjects analyzed signifies those subjects who were evaluable for this endpoint.

The Clinical Global Impression (CGI) scale consists of a 7-point clinician rating of illness severity (1 = normal, not at all ill, 7 = among the most extremely ill patients), at the beginning of the trial (baseline) - Clinical Global Impression-Severity Rating (CGI-S) and a 7-point clinician rating of improvement of patient condition (1=very much improved since baseline/initiation of treatment, 7=very much worse from baseline), during and at the end of the trial (Day 15) - CGI-I. The CGI-I score at Day 15 is presented for this outcome measure.

Time frame: At Day 15

Population: The analysis population included the full analysis set (FAS) which consisted of all subjects in the safety population who had the primary efficacy endpoint measured (at baseline and phone call \[Day 8\] ±1 or Day 15). Analyses for the FAS were conducted according to the randomized treatment.~Number of subjects analyzed signifies those subjects who were evaluable for this endpoint.