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Completed

NCT Number: NCT02034071

Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome

• 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.

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Key information

Conditions

Prader-Willi Syndrome Abnormalities, Multiple Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Imprinting Disorders Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutrition Disorders Nutritional and Metabolic Diseases Obesity Overnutrition Overweight

Age range

10 year–22 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

University of California, Irvine

Orange, California, 92686, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

DCCR

Drug

Other names: Diazoxide Choline Controlled-Release Tablet

Placebo

Drug

Primary outcomes

  1. Hyperphagia using hyperphagia questionnaire

    Time frame: Change from Day 69 through Day 97

  2. Resting energy expenditure

    Time frame: Change from Day 69 through Day 97

Other outcomes

  1. Weight

    Time frame: Percent Change from Baseline through Day 69

  2. Weight

    Time frame: Percent Change from Day 69 through Day 97

  3. Resting energy expenditure

    Time frame: Change from Baseline through Day 69

  4. Hyperphagia using hyperphagia questionnaire

    Time frame: Change from Baseline through Day 69

  5. Percent Body Fat

    Time frame: Change from Baseline through Day 69

  6. Percent Body Fat

    Time frame: Change from Day 69 through Day 97

  7. Lipids

    Time frame: 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

  8. Lipids

    Time frame: Percent Change from Day 69 Through Day 97

    Percent change from Day 69 through Day 97 for triglycerides, total cholesterol, LDL cholesterol, HDL cholesterol, and non-HDL cholesterol

Sponsors and collaborators

Lead sponsor

Essentialis, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2014
Primary completion
2015
Study completion
2015
First posted
Jan 13, 2014
Registry last updated
Sep 1, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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