Phase 3
N=126
A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT03440814 ↗Enrolled (actual)
126
Serious AEs
4.8%
Results posted
Sep 2023
Primary outcome: Primary: Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) — -5.94; -4.27 score on a scale — p=0.1983
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- DCCR (Drug); Placebo for DCCR (Drug)
- Age
- Pediatric, Adult, Older Adult · 4+ yrs
- Sex
- All
- Sponsor
- Soleno Therapeutics, Inc.
- Primary completion
- May 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) |
-5.94; -4.27 | 0.1983 |
| SECONDARY Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13) |
0; 0; 5; 0; 25; 2 | 0.0294 sig |
| SECONDARY Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13) |
4; 1; 6; 3; 22; 8 | 0.4089 |
| SECONDARY Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13) |
-0.80; 0.25 | 0.0225 sig |
Summary
The purpose of this is study is to evaluate the effects of DCCR (diazoxide choline controlled release tablets) in children and adults with Prader-Willi syndrome.
Eligibility Criteria
Inclusion Criteria
- Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (patients, as appropriate)
- Genetically-confirmed Prader-Willi syndrome and hyperphagic
- In a stable care setting for at least 6 months prior to Visit 1
- Caregiver must have been caring for the patient for at least 6 months prior to Visit 1
Exclusion Criteria
- Have participated in an interventional clinical study (i.e., investigational drug or device, approved drugs or device evaluated for unapproved use) within prior 3 months
- Positive urine pregnancy test (in females of child-bearing potential) or females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation
- Any other known disease and/or condition, which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol
Data sourced from ClinicalTrials.gov (NCT03440814). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.