Trial · NCT03440814
A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome
NCT03440814, Phase 3, Completed, Soleno Therapeutics, Inc.; results first posted 2023-09-21
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome. Trials, record NCT03440814. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03440814
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome
- NCT number
- NCT03440814
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 127 participants, actual
- Lead sponsor
- Soleno Therapeutics, Inc.
- Conditions
- Prader-Willi Syndrome
- Interventions
- DCCR, Placebo for DCCR
- First posted
- 2018-02-22
- Last updated
- 2023-09-21
- Results first posted
- 2023-09-21
- Limitations and caveats
All subjects were enrolled and had baseline visits prior to the COVID-19 pandemic; however, there was a subset of subjects who needed to complete some of their visits after the onset of the pandemic. The pandemic forced numerous restrictions on normal activities and involved significant disruptions, which had a substantially adverse but inconsistent effect on the study conduct and results. For this reason, the ITT population - Pre-COVID analysis includes all data through March 1, 2020.
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) | Baseline to Visit 7 (Week 13) | 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. |
| Secondary | Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) | at Visit 7 (Week 13) | 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. |
| Secondary | Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13) | at Visit 7 (Week 13) | 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. |
| Secondary | Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13) | Baseline to Visit 7 (Week 13) | 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). |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) | Baseline to Visit 7 (Week 13) | 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. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) | at Visit 7 (Week 13) | 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 | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13) | at Visit 7 (Week 13) | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13) | Baseline to Visit 7 (Week 13) | 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. | kg | LEAST_SQUARES_MEAN |
| POST_HOC | Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) - Pre-COVID Analysis | Baseline to Visit 7 (Week 13) | 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. | score on a scale | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group34
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) | DCCR | -5.94 | Standard Error 0.879 | score on a scale | |
| Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) | Placebo for DCCR | -4.27 | Standard Error 1.145 | score on a scale | |
| Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) | DCCR | 1 = Very Much Improved | 0 | Participants | |
| Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) | Placebo for DCCR | 1 = Very Much Improved | 0 | Participants | |
| Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) | DCCR | 2 = Much improved | 5 | Participants | |
| Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) | Placebo for DCCR | 2 = Much improved | 0 | Participants | |
| Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) | DCCR | 3 = Minimally improved | 25 | Participants | |
| Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) | Placebo for DCCR | 3 = Minimally improved | 2 | Participants | |
| Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) | DCCR | 4 = No change | 41 | Participants | |
| Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) | Placebo for DCCR | 4 = No change | 37 | Participants |
1 to 10 of 34Source ClinicalTrials.gov
See all 34 results by groupParticipant flow10
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | DCCR | 84 | |
| Overall Study | STARTED | Placebo for DCCR | 42 | |
| Overall Study | Randomized (May or May Not Have Taken Study Drug) | DCCR | 85 | |
| Overall Study | Randomized (May or May Not Have Taken Study Drug) | Placebo for DCCR | 42 | |
| Overall Study | Intent-to-Treat (ITT) Population | DCCR | 82 | |
| Overall Study | Intent-to-Treat (ITT) Population | Placebo for DCCR | 42 | |
| Overall Study | COMPLETED | DCCR | 79 | |
| Overall Study | COMPLETED | Placebo for DCCR | 41 | |
| Overall Study | NOT COMPLETED | DCCR | 5 | |
| Overall Study | NOT COMPLETED | Placebo for DCCR | 1 |
Source ClinicalTrials.gov
Adverse events38
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Hypertrichosis | Skin and subcutaneous tissue disorders | DCCR | 30 | 84 | 32 |
| other | Hypertrichosis | Skin and subcutaneous tissue disorders | Placebo for DCCR | 6 | 42 | 6 |
| other | Hirsutism | Skin and subcutaneous tissue disorders | DCCR | 6 | 84 | 8 |
| other | Hirsutism | Skin and subcutaneous tissue disorders | Placebo for DCCR | 3 | 42 | 6 |
| other | Upper respiratory tract infection | Infections and infestations | DCCR | 9 | 84 | 9 |
| other | Upper respiratory tract infection | Infections and infestations | Placebo for DCCR | 5 | 42 | 7 |
| other | Oedema peripheral | General disorders | DCCR | 16 | 84 | 28 |
| other | Oedema peripheral | General disorders | Placebo for DCCR | 4 | 42 | 4 |
| other | Pyrexia | General disorders | DCCR | 5 | 84 | 6 |
| other | Pyrexia | General disorders | Placebo for DCCR | 0 | 42 | 0 |
1 to 10 of 38Source ClinicalTrials.gov
See all 38 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DCCR | 0 | 84 |
| Total, all-cause mortality | Placebo for DCCR | 0 | 42 |
| Total, other adverse events | DCCR | 69 | 84 |
| Total, other adverse events | Placebo for DCCR | 31 | 42 |
| Total, serious adverse events | DCCR | 6 | 84 |
| Total, serious adverse events | Placebo for DCCR | 0 | 42 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| DCCR vs Placebo for DCCR: deaths from any cause | Trial comparisons |
| DCCR vs Placebo for DCCR: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03440814
GET https://conductscience.org/api/trials/v1/NCT03440814/results?page=2