Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
Completed · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Prader-Willi Syndrome
In brief
• This is a single-center, open-label, single-arm study with a double-blind, placebo-controlled, randomized withdrawal extension. Patients are initiated on a DCCR dose of about 1.5 mg/kg (maximum starting dose of 145 mg) and are titrated every 14 days to about 2.4 mg/kg, 3.3 mg/kg, 4.2 mg/kg, and 5.1 mg/kg (maximum dose of 507.5 mg). These DCCR doses are equivalent to diazoxide doses of 1.03, 1.66, 2.28, 2.9, and 3.52 mg/kg. The administered dose will be as close to the mg/kg dosing as can be achieved by the available dose strengths of DCCR. Patients will be up-titrated at each visit at the discretion of the investigator. All patients will be continued in the double-blind, placebo-controlled, randomized withdrawal extension. Any patient who showed an increase in resting energy expenditure and/or a reduction in hyperphagia from Baseline through Day 55 or Day 69 will be designated a responder, whereas all others will be designated non-responders. Responders will be randomized in a 1:1 ratio either to continue on active treatment at the dose they were treated with on Day 69 or to the placebo equivalent of that dose for an additional 4 weeks. Non-responders will continue open label treatment during the extension.
Key facts
- Study ID
- NCT02034071
- Run by
- Essentialis, Inc.
- People needed
- 13
- Starts
- 2014-04-01
- Expected to finish
- 2015-05-01
- Last updated by the study team
- 2016-09-01
Who can join
Age: 10 and older, up to 22. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Children. adolescents and young adults with genetically confirmed Prader-Willi syndrome
- Ages at ≥ 10 years and ≤ 22 years
- Generally healthy as documented by the medical history, physical examination, vital sign assessments, 12-lead electrocardiogram (ECG), and clinical laboratory assessments
- BMI exceeds the 95th percentile of the age specific BMI value on the CDC BMI charts
- Fasting glucose ≤ 126 mg/dL
- HbA1c ≤ 6.5 %
You may not qualify if…
- Administration of investigational drugs within 1 month prior to Screening Visit
- Anticipated requirement for use of prohibited medications
- History of allergic reaction or significant intolerance to: diazoxide, thiazides or sulfonamides
- Anticipate transitions in their care from family home to group home or other similar potentially disruptive changes
- Congestive heart failure or known compromised cardiac reserve
- Any other clinically significant endocrine, cardiovascular, pulmonary, neurological, psychiatric, hepatic, gastrointestinal, hematological, renal, or dermatological disease interfering with the assessments of the investigational drug, according to the Investigator
Where it is running
- University of California, Irvine — Orange, California, United States
Full record on ClinicalTrials.gov
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