Open-Label Extension Study of Diazoxide Choline in Patients With Prader-Willi Syndrome
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT03714373 |
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Recruitment Status :
Active, not recruiting
First Posted : October 22, 2018
Last Update Posted : June 18, 2020
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Sponsor:
Soleno Therapeutics, Inc.
Information provided by (Responsible Party):
Soleno Therapeutics, Inc.
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Brief Summary:
The purpose of this is study is to evaluate the long term safety of DCCR (diazoxide choline controlled release tablets) in children and adults with Prader-Willi syndrome. After completion of 1 year in this study, subjects will have the option to continue treatment for up to 2 additional years.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Prader-Willi Syndrome | Drug: DCCR | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 105 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | An Open-Label, Long-Term Safety Evaluation of Diazoxide Choline Controlled-Release Tablet in Patients With Prader-Willi Syndrome |
| Actual Study Start Date : | October 1, 2018 |
| Estimated Primary Completion Date : | April 2021 |
| Estimated Study Completion Date : | June 2023 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Prader-Willi syndrome
MedlinePlus related topics:
Prader-Willi Syndrome
| Arm | Intervention/treatment |
|---|---|
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Experimental: DCCR
75 - 450 mg DCCR
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Drug: DCCR
Once daily oral administration |
Primary Outcome Measures :
- Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported [ Time Frame: Baseline to Week 54 or until resolution of certain adverse events at the discretion of the Investigator and Sponsor ]Safety analyses will be conducted in all subjects who receive at least one dose of DCCR. Adverse events will be described by type and level of severity.
Secondary Outcome Measures :
- Hyperphagia Score [ Time Frame: Baseline to Week 52 ]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.
- Body fat mass (DXA) [ Time Frame: Baseline at Week 52 ]Change in Body Fat Mass from Baseline to Week 52
- Clinical Global Impression of Improvement (CGI-I) [ Time Frame: at Week 52 ]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 provides 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 (best to worst).
- Caregiver Global Impression of Change (Caregiver GI-C) [ Time Frame: at Week 52 ]The Caregiver 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 provides 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 (best to worst).
Information from the National Library of Medicine
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| Ages Eligible for Study: | 4 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Key Inclusion Criteria:
- Successful completion of clinical study C601
- Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (subjects, as appropriate)
Key Exclusion Criteria:
- 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 90 days after study participation
- Any new disease, condition, or circumstance which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol (e.g., an anticipated change of care setting)
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03714373
Locations
Show 28 study locations
Sponsors and Collaborators
Soleno Therapeutics, Inc.
| Responsible Party: | Soleno Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT03714373 |
| Other Study ID Numbers: |
C602 |
| First Posted: | October 22, 2018 Key Record Dates |
| Last Update Posted: | June 18, 2020 |
| Last Verified: | June 2020 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Soleno Therapeutics, Inc.:
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PWS Prader-Willi Syndrome |
Additional relevant MeSH terms:
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Prader-Willi Syndrome Syndrome Disease Pathologic Processes Intellectual Disability Neurobehavioral Manifestations Neurologic Manifestations Nervous System Diseases |
Abnormalities, Multiple Congenital Abnormalities Chromosome Disorders Genetic Diseases, Inborn Obesity Overnutrition Nutrition Disorders |