Trial record 8 of 37 for:    "Prader-Willi syndrome"

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by Essentialis, Inc.
Sponsor:
Information provided by (Responsible Party):
Essentialis, Inc.
ClinicalTrials.gov Identifier:
NCT02034071
First received: January 8, 2014
Last updated: May 16, 2014
Last verified: May 2014
  Purpose

• 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.


Condition Intervention Phase
Prader-Willi Syndrome
Drug: DCCR
Drug: Placebo
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by Essentialis, Inc.:

Primary Outcome Measures:
  • Hyperphagia using hyperphagia questionnaire [ Time Frame: Change from Day 69 through Day 97 ] [ Designated as safety issue: No ]
  • Resting energy expenditure [ Time Frame: Change from Day 69 through Day 97 ] [ Designated as safety issue: No ]

Other Outcome Measures:
  • Weight [ Time Frame: Percent Change from Baseline through Day 69 ] [ Designated as safety issue: No ]
  • Weight [ Time Frame: Percent Change from Day 69 through Day 97 ] [ Designated as safety issue: No ]
  • Resting energy expenditure [ Time Frame: Change from Baseline through Day 69 ] [ Designated as safety issue: No ]
  • Hyperphagia using hyperphagia questionnaire [ Time Frame: Change from Baseline through Day 69 ] [ Designated as safety issue: No ]
  • Percent Body Fat [ Time Frame: Change from Baseline through Day 69 ] [ Designated as safety issue: No ]
  • Percent Body Fat [ Time Frame: Change from Day 69 through Day 97 ] [ Designated as safety issue: No ]
  • Lipids [ Time Frame: Percent Change from Baseline through Day 69 ] [ Designated as safety issue: No ]
    Percent change from Baseline through Day 69 for triglycerides, total cholesterol, LDL cholesterol, HDL cholesterol, and non-HDL cholesterol

  • Lipids [ Time Frame: Percent Change from Day 69 Through Day 97 ] [ Designated as safety issue: No ]
    Percent change from Day 69 through Day 97 for triglycerides, total cholesterol, LDL cholesterol, HDL cholesterol, and non-HDL cholesterol


Estimated Enrollment: 12
Study Start Date: April 2014
Estimated Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   10 Years to 20 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02034071

Contacts
Contact: Marie Wencel, Clinical Research Coordinator (949) 824-0521 mwencel@uci.edu

Locations
United States, California
University of California, Irvine Recruiting
Orange, California, United States, 92686
Contact: Virginia Kimonis, MD    714-456-2942    vkimonis@uci.edu   
Principal Investigator: Virginia Kimonis, MD         
Sponsors and Collaborators
Essentialis, Inc.
Investigators
Principal Investigator: Virginia Kimonis, MD University of California, Irvine
  More Information

Additional Information:
No publications provided

Responsible Party: Essentialis, Inc.
ClinicalTrials.gov Identifier: NCT02034071     History of Changes
Other Study ID Numbers: PC025
Study First Received: January 8, 2014
Last Updated: May 16, 2014
Health Authority: United States: Food and Drug Administration

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

Additional relevant MeSH terms:
Prader-Willi Syndrome
Mental Retardation
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
Pharmacologic Actions
Gastrointestinal Agents
Therapeutic Uses
Lipid Regulating Agents
Nootropic Agents
Central Nervous System Agents
Antihypertensive Agents
Cardiovascular Agents
Vasodilator Agents

ClinicalTrials.gov processed this record on July 22, 2014