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

Primary Purpose

Prader-Willi Syndrome

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
DCCR
Placebo
Sponsored by
Essentialis, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Prader-Willi Syndrome focused on measuring Prader-Willi syndrome

Eligibility Criteria

10 Years - 22 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of California, Irvine

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

DCCR Open Label - DCCR Double Blind

DCCR Open Label - Placebo Double Blind

Arm Description

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

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

Outcomes

Primary Outcome Measures

Hyperphagia using hyperphagia questionnaire
Resting energy expenditure

Secondary Outcome Measures

Full Information

First Posted
January 8, 2014
Last Updated
August 30, 2016
Sponsor
Essentialis, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT02034071
Brief Title
Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
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 Type
Interventional

2. Study Status

Record Verification Date
August 2016
Overall Recruitment Status
Completed
Study Start Date
April 2014 (undefined)
Primary Completion Date
May 2015 (Actual)
Study Completion Date
May 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Essentialis, Inc.

4. Oversight

Data Monitoring Committee
Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome
Keywords
Prader-Willi syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
13 (Actual)

8. Arms, Groups, and Interventions

Arm Title
DCCR Open Label - DCCR Double Blind
Arm Type
Experimental
Arm Description
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
Arm Title
DCCR Open Label - Placebo Double Blind
Arm Type
Experimental
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
DCCR
Other Intervention Name(s)
Diazoxide Choline Controlled-Release Tablet
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Hyperphagia using hyperphagia questionnaire
Time Frame
Change from Day 69 through Day 97
Title
Resting energy expenditure
Time Frame
Change from Day 69 through Day 97
Other Pre-specified Outcome Measures:
Title
Weight
Time Frame
Percent Change from Baseline through Day 69
Title
Weight
Time Frame
Percent Change from Day 69 through Day 97
Title
Resting energy expenditure
Time Frame
Change from Baseline through Day 69
Title
Hyperphagia using hyperphagia questionnaire
Time Frame
Change from Baseline through Day 69
Title
Percent Body Fat
Time Frame
Change from Baseline through Day 69
Title
Percent Body Fat
Time Frame
Change from Day 69 through Day 97
Title
Lipids
Description
Percent change from Baseline through Day 69 for triglycerides, total cholesterol, LDL cholesterol, HDL cholesterol, and non-HDL cholesterol
Time Frame
Percent Change from Baseline through Day 69
Title
Lipids
Description
Percent change from Day 69 through Day 97 for triglycerides, total cholesterol, LDL cholesterol, HDL cholesterol, and non-HDL cholesterol
Time Frame
Percent Change from Day 69 Through Day 97

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
22 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Virginia Kimonis, MD
Organizational Affiliation
University of California, Irvine
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of California, Irvine
City
Orange
State/Province
California
ZIP/Postal Code
92686
Country
United States

12. IPD Sharing Statement

Links:
URL
http://www.pwsausa.org/
Description
Prader-Willi Syndrome Association USA
URL
http://ghr.nlm.nih.gov/condition/prader-willi-syndrome
Description
Genetics Home Reference - Prader-Willi syndrome
URL
http://www.fpwr.org/about-prader-willi-syndrome
Description
Foundation for Prader-Willi Research - About Prader-Willi syndrome

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

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