Prader-Willi Syndrome
Conditions
Keywords
Prader-Willi syndrome
Brief summary
• 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.
Interventions
DRUGDCCR
DRUGPlacebo
Sponsors
Essentialis, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
10 Years to 22 Years
Healthy volunteers
No
Inclusion criteria
* 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 %
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hyperphagia using hyperphagia questionnaire | Change from Day 69 through Day 97 |
| Resting energy expenditure | Change from Day 69 through Day 97 |
Other
| Measure | Time frame | Description |
|---|---|---|
| Hyperphagia using hyperphagia questionnaire | Change from Baseline through Day 69 | — |
| Weight | Percent Change from Baseline through Day 69 | — |
| Lipids | Percent Change from Baseline through Day 69 | Percent change from Baseline through Day 69 for triglycerides, total cholesterol, LDL cholesterol, HDL cholesterol, and non-HDL cholesterol |
| Percent Body Fat | Change from Baseline through Day 69 | — |
| Resting energy expenditure | Change from Baseline through Day 69 | — |
Countries
United States
Outcome results
None listed