Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
| Status: | Recruiting |
|---|---|
| Conditions: | Women's Studies |
| Therapuetic Areas: | Reproductive |
| Healthy: | No |
| Age Range: | 10 - 20 |
| Updated: | 2/7/2015 |
| Start Date: | April 2014 |
| Contact: | Marie Wencel, Clinical Research Coordinator |
| Email: | mwencel@uci.edu |
| Phone: | (949) 824-0521 |
A Dose Titration Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome With a Double-Blind, Placebo-Controlled, Randomized Withdrawal Extension
• 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 randomized 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. Patients 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.
Randomization will be stratified on responder/non-responder.
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 randomized 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. Patients 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.
Randomization will be stratified on responder/non-responder.
Inclusion Criteria:
- Children. adolescents and young adults with genetically confirmed Prader-Willi
syndrome
- Ages at ≥ 10 years and ≤ 20 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 %
Exclusion Criteria:
- 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
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