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

NCT02034071 | Completed Phase 1 DMC

• 1 other identifier: PC025
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

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.

Conditions

Prader-Willi Syndrome

Keywords

Prader-Willi syndrome

Outcome Measures

Primary Outcomes (2)

• Hyperphagia using hyperphagia questionnaire

  Change from Day 69 through Day 97

• Resting energy expenditure

  Change from Day 69 through Day 97

Other Outcomes (8)

• Weight

  Percent Change from Baseline through Day 69

• Weight

  Percent Change from Day 69 through Day 97

• Resting energy expenditure

  Change from Baseline through Day 69

Study Arms (2)

DCCR Open Label - DCCR Double Blind

EXPERIMENTAL

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

DCCR Open Label - Placebo Double Blind

EXPERIMENTAL

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; Drug: Placebo

Interventions

DCCR DRUG

Also known as: Diazoxide Choline Controlled-Release Tablet

DCCR Open Label - DCCR Double Blind; DCCR Open Label - Placebo Double Blind

Placebo DRUG

DCCR Open Label - Placebo Double Blind

Eligibility Criteria

• Age: 10 Years - 22 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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 %

You may not qualify if:

• 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

Sponsors & Collaborators

• Essentialis, Inc.: lead

Study Sites (1)

University of California, Irvine

Orange, California, 92686, United States

Location

Related Links

• Prader-Willi Syndrome Association USA
• Genetics Home Reference - Prader-Willi syndrome
• Foundation for Prader-Willi Research - About Prader-Willi syndrome

MeSH Terms

Conditions

Prader-Willi Syndrome

Condition Hierarchy (Ancestors)

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

Study Officials

• Virginia Kimonis, MD

  University of California, Irvine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 8, 2014
• First Posted: January 13, 2014
• Study Start: April 1, 2014
• Primary Completion: May 1, 2015
• Study Completion: May 1, 2015
• Last Updated: September 1, 2016

Record last verified: 2016-08

Locations

US (1)