A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome

NCT03440814 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: C601
• Study Type: interventional
• Target: 127
• Locations: 2 countries
• Sites: 29

Brief Summary

The purpose of this is study is to evaluate the effects of DCCR (diazoxide choline controlled release tablets) in children and adults with Prader-Willi syndrome.

Conditions

Prader-Willi Syndrome

Keywords

PWS; Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

• Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13)

  Hyperphagia-related behaviors were assessed by the validated hyperphagia questionnaire for clinical trials (HQ-CT), an instrument designed to measure symptoms of food related preoccupations and behaviors that was completed by the caregiver. The HQ-CT consists of nine items with responses ranging from 0-4 units each (possible total score range: 0-36). The HQ-CT was assessed at Screening, Baseline (Visit 2), and approximately every 4 weeks post-dose at Week 4, Week 8, and Week 13. A decrease in score from baseline represented improvement.

  Baseline to Visit 7 (Week 13)

Secondary Outcomes (3)

• Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13)

  at Visit 7 (Week 13)

• Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13)

  at Visit 7 (Week 13)

• Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13)

  Baseline to Visit 7 (Week 13)

Study Arms (2)

DCCR

EXPERIMENTAL

75 - 450 mg DCCR

Drug: DCCR

Placebo

PLACEBO COMPARATOR

75 - 450 mg placebo for DCCR

Drug: Placebo for DCCR

Interventions

DCCR DRUG

Once daily oral administration

DCCR

Placebo for DCCR DRUG

Once daily oral administration

Placebo

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (patients, as appropriate)
• Genetically-confirmed Prader-Willi syndrome and hyperphagic
• In a stable care setting for at least 6 months prior to Visit 1
• Caregiver must have been caring for the patient for at least 6 months prior to Visit 1

You may not qualify if:

• Have participated in an interventional clinical study (i.e., investigational drug or device, approved drugs or device evaluated for unapproved use) within prior 3 months
• Positive urine pregnancy test (in females of child-bearing potential) or females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation
• Any other known disease and/or condition, which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol

Sponsors & Collaborators

• Soleno Therapeutics, Inc.: lead

Study Sites (29)

University of California, Irvine

Orange, California, 92868, United States

Location

Stanford University

Palo Alto, California, 94305, United States

Location

Rady Children's Hospital San Diego

San Diego, California, 92123, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

University of Florida Gainesville

Gainesville, Florida, 32608, United States

Location

Emory Children's Center

Atlanta, Georgia, 30322, United States

Location

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

Kansas University Medical Center

Kansas City, Kansas, 66160, United States

Location

National Institutes of Health Hatfield Clinical Research Center

Bethesda, Maryland, 20892, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Sparrow Clinical Research Institute

Lansing, Michigan, 48912, United States

Location

Children's Minnesota

Saint Paul, Minnesota, 55102, United States

Location

St. Joseph's University Medical Center

Paterson, New Jersey, 07503, United States

Location

NYU Winthrop Hospital

Mineola, New York, 11501, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

The Research Institute at Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Vanderbilt University

Nashville, Tennessee, 37232, United States

Location

Research Institute of Dallas

Dallas, Texas, 75231, United States

Location

University of Utah

Salt Lake City, Utah, 84113, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

The Queen Elizabeth University

Glasgow, Scottland, G51 4TF, United Kingdom

Location

Hull and East Yorkshire Hospitals NHS Trust

Hull, Yorkshire, HU3 2JZ, United Kingdom

Location

Birmingham Women's and Children's Hospital

Birmingham, B4 6NH, United Kingdom

Location

Fulbourn Hospital

Cambridge, CB21 5ER, United Kingdom

Location

Alder Hey Children's Hospital NHS Foundation Trust

Liverpool, L12 2AP, United Kingdom

Location

Aintree University Hospital NHS Foundation Trust

Liverpool, L9 7AL, United Kingdom

Location

Royal London Hospital

London, E1 1BB, United Kingdom

Location

Chelsea and Westminster Hospital

London, SW10 9NH, United Kingdom

Location

Hammersmith Hospital

London, W12 OHS, United Kingdom

Location

Related Publications (3)

• Strong TV, Miller JL, McCandless SE, Gevers E, Yanovski JA, Matesevac L, Bohonowych J, Ballal S, Yen K, Hirano P, Cowen NM, Bhatnagar A. Behavioral changes in patients with Prader-Willi syndrome receiving diazoxide choline extended-release tablets compared to the PATH for PWS natural history study. J Neurodev Disord. 2024 Apr 26;16(1):22. doi: 10.1186/s11689-024-09536-x.

  PMID: 38671361 DERIVED

• Miller JL, Gevers E, Bridges N, Yanovski JA, Salehi P, Obrynba KS, Felner EI, Bird LM, Shoemaker AH, Angulo M, Butler MG, Stevenson D, Goldstone AP, Wilding J, Lah M, Shaikh MG, Littlejohn E, Abuzzahab MJ, Fleischman A, Hirano P, Yen K, Cowen NM, Bhatnagar A; C601/C602 Investigators. Diazoxide choline extended-release tablet in people with Prader-Willi syndrome: results from long-term open-label study. Obesity (Silver Spring). 2024 Feb;32(2):252-261. doi: 10.1002/oby.23928. Epub 2023 Nov 2.

  PMID: 37919617 DERIVED

• Miller JL, Gevers E, Bridges N, Yanovski JA, Salehi P, Obrynba KS, Felner EI, Bird LM, Shoemaker AH, Angulo M, Butler MG, Stevenson D, Abuzzahab J, Barrett T, Lah M, Littlejohn E, Mathew V, Cowen NM, Bhatnagar A; DESTINY PWS Investigators. Diazoxide Choline Extended-Release Tablet in People With Prader-Willi Syndrome: A Double-Blind, Placebo-Controlled Trial. J Clin Endocrinol Metab. 2023 Jun 16;108(7):1676-1685. doi: 10.1210/clinem/dgad014.

  PMID: 36639249 DERIVED

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

Limitations and Caveats

All subjects were enrolled and had baseline visits prior to the COVID-19 pandemic; however, there was a subset of subjects who needed to complete some of their visits after the onset of the pandemic. The pandemic forced numerous restrictions on normal activities and involved significant disruptions, which had a substantially adverse but inconsistent effect on the study conduct and results. For this reason, the ITT population - Pre-COVID analysis includes all data through March 1, 2020.

Results Point of Contact

• Title: C601 Project Manager
• Organization: Soleno Therapeutics, Inc.

C601ProjectManager@soleno.life

650-353-2051

Study Officials

• Evelien Gevers, MD, PhD

  Queen Mary University of London, Barts Health NHS Trust

  PRINCIPAL INVESTIGATOR

• Jennifer L. Miller, MD

  University of Florida

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 13, 2018
• First Posted: February 22, 2018
• Study Start: May 9, 2018
• Primary Completion: May 1, 2020
• Study Completion: May 1, 2020
• Last Updated: September 21, 2023
• Results First Posted: September 21, 2023

Record last verified: 2023-06

Locations

GB (9); US (20)