Trial Outcomes & Findings for A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome (NCT NCT03440814)

Last Updated: 2023-09-21

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

127 participants

Primary outcome timeframe

Baseline to Visit 7 (Week 13)

Results posted on

2023-09-21

Participant Flow

People with Prader-Willi syndrome (PWS) were recruited for this study. Those who met the eligibility criteria were enrolled / randomized. Caregivers of subjects were responsible for completing the protocol-specified caregiver questionnaires and were not enrolled in this study. The first site in US started recruiting subjects on 17May2018; the first site in UK started recruiting subjects on 26Jun2019; all sites were either hospitals or academic medical centers.

181 subjects were screened, 158 were eligible and entered the two-week, single-blind placebo run-in period and 127 subjects were enrolled / randomized in the trial. 126 of the 127 subjects took any amount of study drug.

Participant milestones

Participant milestones
Measure	DCCR Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg DCCR tablets could be taken; DCCR was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the DCCR treatment group were titrated every 2 weeks until the target dose was achieved.	Placebo for DCCR Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg placebo for DCCR tablets could be taken; Placebo was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the Placebo group were titrated on placebo, similar to the DCCR group.
Overall Study STARTED	84	42
Overall Study Randomized (May or May Not Have Taken Study Drug)	85	42
Overall Study Intent-to-Treat (ITT) Population	82	42
Overall Study COMPLETED	79	41
Overall Study NOT COMPLETED	5	1

Reasons for withdrawal

Reasons for withdrawal
Measure	DCCR Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg DCCR tablets could be taken; DCCR was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the DCCR treatment group were titrated every 2 weeks until the target dose was achieved.	Placebo for DCCR Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg placebo for DCCR tablets could be taken; Placebo was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the Placebo group were titrated on placebo, similar to the DCCR group.
Overall Study Adverse Event	2	1
Overall Study Lost to Follow-up	2	0
Overall Study Consent withdrawn by parent/legal guardian	1	0

Baseline Characteristics

A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	DCCR n=84 Participants Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg DCCR tablets could be taken; DCCR was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the DCCR treatment group were titrated every 2 weeks until the target dose was achieved.	Placebo for DCCR n=42 Participants Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg placebo for DCCR tablets could be taken; Placebo was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the Placebo group were titrated on placebo, similar to the DCCR group.	Total n=126 Participants Total of all reporting groups
Age, Continuous	13.2 years n=93 Participants	13.6 years n=4 Participants	13.4 years n=27 Participants
Age, Customized <8 years	18 Participants n=93 Participants	7 Participants n=4 Participants	25 Participants n=27 Participants
Age, Customized 8 to <12 years	21 Participants n=93 Participants	14 Participants n=4 Participants	35 Participants n=27 Participants
Age, Customized 12 to <18 years	30 Participants n=93 Participants	11 Participants n=4 Participants	41 Participants n=27 Participants
Age, Customized 18 to <65 years	15 Participants n=93 Participants	10 Participants n=4 Participants	25 Participants n=27 Participants
Age, Customized >=65 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Sex: Female, Male Female	47 Participants n=93 Participants	23 Participants n=4 Participants	70 Participants n=27 Participants
Sex: Female, Male Male	37 Participants n=93 Participants	19 Participants n=4 Participants	56 Participants n=27 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=93 Participants	7 Participants n=4 Participants	13 Participants n=27 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	77 Participants n=93 Participants	34 Participants n=4 Participants	111 Participants n=27 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=93 Participants	1 Participants n=4 Participants	2 Participants n=27 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Race/Ethnicity, Customized Asian	0 Participants n=93 Participants	1 Participants n=4 Participants	1 Participants n=27 Participants
Race/Ethnicity, Customized Native Hawaiian or Pacific Islander	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Race/Ethnicity, Customized Black or African American	4 Participants n=93 Participants	2 Participants n=4 Participants	6 Participants n=27 Participants
Race/Ethnicity, Customized White	72 Participants n=93 Participants	34 Participants n=4 Participants	106 Participants n=27 Participants
Race/Ethnicity, Customized Multiple	5 Participants n=93 Participants	3 Participants n=4 Participants	8 Participants n=27 Participants
Race/Ethnicity, Customized Other	1 Participants n=93 Participants	2 Participants n=4 Participants	3 Participants n=27 Participants
HQ-CT Total Score	23.0 score on a scale STANDARD_DEVIATION 6.01 • n=93 Participants	21.9 score on a scale STANDARD_DEVIATION 5.08 • n=4 Participants	22.7 score on a scale STANDARD_DEVIATION 5.72 • n=27 Participants

PRIMARY outcome

Timeframe: Baseline to Visit 7 (Week 13)

Population: Intent to treat (ITT) population includes all randomized subjects who had a Baseline HQ-CT value and at least one post-Baseline HQ-CT value.

Outcome measures

Outcome measures
Measure	DCCR n=82 Participants Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg DCCR tablets could be taken; DCCR was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the DCCR treatment group were titrated every 2 weeks until the target dose was achieved.	Placebo for DCCR n=42 Participants Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg placebo for DCCR tablets could be taken; Placebo was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the Placebo group were titrated on placebo, similar to the DCCR group.
Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13)	-5.94 score on a scale Standard Error 0.879	-4.27 score on a scale Standard Error 1.145

SECONDARY outcome

Timeframe: at Visit 7 (Week 13)

Population: Intent to treat (ITT) population includes all randomized subjects who had a Baseline HQ-CT value and at least one post-Baseline HQ-CT value

The Clinical Global Impression of Improvement (CGI-I) is a single statement designed to assess the Investigator's overall perception of change in the subject's condition across the course of the clinical trial. The Investigator provided a response to "Compared to the subject's condition at enrollment, the subject's condition is:" by rating the subject's behavior using a 7-point response scale: Very much improved, Much improved, Minimally improved, No change, Minimally worse, Much worse, and Very much worse. The Investigator only took into account the subject's PWS condition.

Outcome measures

Outcome measures
Measure	DCCR n=82 Participants Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg DCCR tablets could be taken; DCCR was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the DCCR treatment group were titrated every 2 weeks until the target dose was achieved.	Placebo for DCCR n=42 Participants Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg placebo for DCCR tablets could be taken; Placebo was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the Placebo group were titrated on placebo, similar to the DCCR group.
Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) 1 = Very Much Improved	0 Participants	0 Participants
Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) 2 = Much improved	5 Participants	0 Participants
Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) 3 = Minimally improved	25 Participants	2 Participants
Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) 4 = No change	41 Participants	37 Participants
Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) 5 = Minimally worse	11 Participants	3 Participants
Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) 6 = Much worse	0 Participants	0 Participants
Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) 7 = Very much worse	0 Participants	0 Participants

SECONDARY outcome

Timeframe: at Visit 7 (Week 13)

Population: Intent to treat (ITT) population includes all randomized subjects who had a Baseline HQ-CT value and at least one post-Baseline HQ-CT value.

The Caregiver Global Impression of Change (GI-C) is a single statement designed to assess the caregiver's overall perception of change in the subject across the course of the clinical trial. The caregiver provided a response to "Please choose the response below that best describes the overall change in the person's PWS since they started taking the study medication" using a 7-point graded response scale: Very much better, Moderately better, A little better, No change, A little worse, Moderately worse, and Very much worse.

Outcome measures

Outcome measures
Measure	DCCR n=82 Participants Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg DCCR tablets could be taken; DCCR was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the DCCR treatment group were titrated every 2 weeks until the target dose was achieved.	Placebo for DCCR n=42 Participants Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg placebo for DCCR tablets could be taken; Placebo was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the Placebo group were titrated on placebo, similar to the DCCR group.
Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13) 5 = Minimally worse	6 Participants	5 Participants
Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13) 6 = Much worse	4 Participants	3 Participants
Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13) 7 = Very much worse	1 Participants	2 Participants
Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13) 1 = Very Much Improved	4 Participants	1 Participants
Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13) 2 = Much improved	6 Participants	3 Participants
Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13) 3 = Minimally improved	22 Participants	8 Participants
Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13) 4 = No change	39 Participants	20 Participants

SECONDARY outcome

Timeframe: Baseline to Visit 7 (Week 13)

Population: Intent to treat (ITT) population includes all randomized subjects who had a Baseline HQ-CT value and at least one post-Baseline HQ-CT value.

Whole body scans were performed. Reports included a breakdown of the following regions: left arm, right arm, trunk, left leg, right leg, and head. Each region was evaluated for body fat mass (g).

Outcome measures

Outcome measures
Measure	DCCR n=82 Participants Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg DCCR tablets could be taken; DCCR was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the DCCR treatment group were titrated every 2 weeks until the target dose was achieved.	Placebo for DCCR n=42 Participants Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg placebo for DCCR tablets could be taken; Placebo was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the Placebo group were titrated on placebo, similar to the DCCR group.
Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13)	-0.80 kg Standard Error 0.356	0.25 kg Standard Error 0.444

POST_HOC outcome

Timeframe: Baseline to Visit 7 (Week 13)

Population: The pre-COVID analysis population consisted of all subjects who had at least one post-baseline assessment of HQ-CT prior to March 1, 2020, when a national emergency was declared in the US as a result of the COVID-19 pandemic.

Outcome measures

Outcome measures
Measure	DCCR n=82 Participants Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg DCCR tablets could be taken; DCCR was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the DCCR treatment group were titrated every 2 weeks until the target dose was achieved.	Placebo for DCCR n=42 Participants Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg placebo for DCCR tablets could be taken; Placebo was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the Placebo group were titrated on placebo, similar to the DCCR group.
Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) - Pre-COVID Analysis	-6.64 score on a scale Standard Error 1.001	-3.51 score on a scale Standard Error 1.278

Adverse Events

DCCR

Serious events: 6 serious events

Other events: 69 other events

Deaths: 0 deaths

Placebo for DCCR

Serious events: 0 serious events

Other events: 31 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	DCCR n=84 participants at risk Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg DCCR tablets could be taken; DCCR was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the DCCR treatment group were titrated every 2 weeks until the target dose was achieved.	Placebo for DCCR n=42 participants at risk Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg placebo for DCCR tablets could be taken; Placebo was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the Placebo group were titrated on placebo, similar to the DCCR group.
Infections and infestations Pneumonia	2.4% 2/84 • Number of events 2 • Baseline to Visit 7 (Week 13)	0.00% 0/42 • Baseline to Visit 7 (Week 13)
Infections and infestations Lower respiratory tract infection	1.2% 1/84 • Number of events 1 • Baseline to Visit 7 (Week 13)	0.00% 0/42 • Baseline to Visit 7 (Week 13)
Infections and infestations Staphylococcal infection	1.2% 1/84 • Number of events 1 • Baseline to Visit 7 (Week 13)	0.00% 0/42 • Baseline to Visit 7 (Week 13)
Psychiatric disorders Abnormal behaviour	1.2% 1/84 • Number of events 1 • Baseline to Visit 7 (Week 13)	0.00% 0/42 • Baseline to Visit 7 (Week 13)
Psychiatric disorders Aggression	1.2% 1/84 • Number of events 1 • Baseline to Visit 7 (Week 13)	0.00% 0/42 • Baseline to Visit 7 (Week 13)
Respiratory, thoracic and mediastinal disorders Pulmonary oedema	1.2% 1/84 • Number of events 1 • Baseline to Visit 7 (Week 13)	0.00% 0/42 • Baseline to Visit 7 (Week 13)
Respiratory, thoracic and mediastinal disorders Respiratory failure	1.2% 1/84 • Number of events 1 • Baseline to Visit 7 (Week 13)	0.00% 0/42 • Baseline to Visit 7 (Week 13)
General disorders Oedema peripheral	1.2% 1/84 • Number of events 1 • Baseline to Visit 7 (Week 13)	0.00% 0/42 • Baseline to Visit 7 (Week 13)
Skin and subcutaneous tissue disorders Erythema multiforme	1.2% 1/84 • Number of events 1 • Baseline to Visit 7 (Week 13)	0.00% 0/42 • Baseline to Visit 7 (Week 13)

Other adverse events

Other adverse events
Measure	DCCR n=84 participants at risk Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg DCCR tablets could be taken; DCCR was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the DCCR treatment group were titrated every 2 weeks until the target dose was achieved.	Placebo for DCCR n=42 participants at risk Participants with PWS were dosed dependent on their weight; 25mg, 75mg or 150mg placebo for DCCR tablets could be taken; Placebo was taken daily. Tablets must be swallowed whole and should not be broken, crushed or chewed. The target dose by weight (kg) was as follows: Subjects weighing 20 to \< 30 kg took 100mg DCCR/day; Subjects weighing ≥ 30 to \< 40 took 150mg DCCR/day; Subjects weighing ≥ 40 to \< 65 took 225mg DCCR/day; Subjects weighing ≥ 65 to \< 100 took 375mg DCCR/day; Subjects weighing ≥ 100 to \< 135 took 450mg DCCR/day. Subjects randomized to the Placebo group were titrated on placebo, similar to the DCCR group.
Skin and subcutaneous tissue disorders Hypertrichosis	35.7% 30/84 • Number of events 32 • Baseline to Visit 7 (Week 13)	14.3% 6/42 • Number of events 6 • Baseline to Visit 7 (Week 13)
Skin and subcutaneous tissue disorders Hirsutism	7.1% 6/84 • Number of events 8 • Baseline to Visit 7 (Week 13)	7.1% 3/42 • Number of events 6 • Baseline to Visit 7 (Week 13)
Infections and infestations Upper respiratory tract infection	10.7% 9/84 • Number of events 9 • Baseline to Visit 7 (Week 13)	11.9% 5/42 • Number of events 7 • Baseline to Visit 7 (Week 13)
General disorders Oedema peripheral	19.0% 16/84 • Number of events 28 • Baseline to Visit 7 (Week 13)	9.5% 4/42 • Number of events 4 • Baseline to Visit 7 (Week 13)
General disorders Pyrexia	6.0% 5/84 • Number of events 6 • Baseline to Visit 7 (Week 13)	0.00% 0/42 • Baseline to Visit 7 (Week 13)
Gastrointestinal disorders Diarrhoea	1.2% 1/84 • Number of events 1 • Baseline to Visit 7 (Week 13)	9.5% 4/42 • Number of events 4 • Baseline to Visit 7 (Week 13)
Gastrointestinal disorders Abdominal pain	1.2% 1/84 • Number of events 1 • Baseline to Visit 7 (Week 13)	7.1% 3/42 • Number of events 5 • Baseline to Visit 7 (Week 13)
Nervous system disorders Headache	6.0% 5/84 • Number of events 5 • Baseline to Visit 7 (Week 13)	14.3% 6/42 • Number of events 14 • Baseline to Visit 7 (Week 13)
Investigations Blood glucose increased	6.0% 5/84 • Number of events 5 • Baseline to Visit 7 (Week 13)	4.8% 2/42 • Number of events 2 • Baseline to Visit 7 (Week 13)
Metabolism and nutrition disorders Hyperglycaemia	11.9% 10/84 • Number of events 10 • Baseline to Visit 7 (Week 13)	0.00% 0/42 • Baseline to Visit 7 (Week 13)

Additional Information

C601 Project Manager

Soleno Therapeutics, Inc.

Phone: 650-353-2051

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee To avoid disclosures that could jeopardize proprietary rights, ensure accuracy of the study data, and ensure integrity is maintained, PI must submit all manuscripts, abstracts, and presentations related to this study to the Soleno for review at least 45 days before their release or as specified in separate written agreements.
Publication restrictions are in place

Restriction type: OTHER