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

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ClinicalTrials.gov Identifier: NCT02034071
Recruitment Status : Completed
First Posted : January 13, 2014
Last Update Posted : September 1, 2016
Sponsor:
Information provided by (Responsible Party):

Study Description
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.

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: DCCR Drug: Placebo Phase 1 Phase 2

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Study Start Date : April 2014
Primary Completion Date : May 2015
Study Completion Date : May 2015


Arms and Interventions

Arm Intervention/treatment
Experimental: DCCR Open Label - DCCR Double Blind
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 through 4 dose levels of DCCR. Patients will be up-titrated at each visit at the discretion of the investigator. Randomized to continue DCCR, at the same dose as they received on Day 69, in the Double-Blind, Placebo-Controlled, Randomized Withdrawal Extension
Drug: DCCR
Other Name: Diazoxide Choline Controlled-Release Tablet
Experimental: DCCR Open Label - Placebo Double Blind
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 through 4 dose levels of DCCR. Patients will be up-titrated at each visit at the discretion of the investigator. Randomized to receive placebo equivalent to the DCCR dose received on Day 69 in the Double-Blind, Placebo-Controlled, Randomized Withdrawal Extension
Drug: DCCR
Other Name: Diazoxide Choline Controlled-Release Tablet
Drug: Placebo


Outcome Measures

Primary Outcome Measures :
  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 Outcome Measures:
  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


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years to 22 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations

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): NCT02034071


Locations
United States, California
University of California, Irvine
Orange, California, United States, 92686
Sponsors and Collaborators
Essentialis, Inc.
Investigators
Principal Investigator: Virginia Kimonis, MD University of California, Irvine
More Information

Additional Information:
Responsible Party: Essentialis, Inc.
ClinicalTrials.gov Identifier: NCT02034071     History of Changes
Other Study ID Numbers: PC025
First Posted: January 13, 2014    Key Record Dates
Last Update Posted: September 1, 2016
Last Verified: August 2016

Keywords provided by Essentialis, Inc.:
Prader-Willi syndrome

Additional relevant MeSH terms:
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
Choline
Diazoxide
Lipotropic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Gastrointestinal Agents
Lipid Regulating Agents
Nootropic Agents
Antihypertensive Agents
Vasodilator Agents