Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period

NCT03714373 | Completed Phase 3 FDA Drug

• 1 other identifier: C602
• Study Type: interventional
• Target: 115
• Locations: 2 countries
• Sites: 28

Brief Summary

This is a multi-center, multi-period study with an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period evaluating the safety and efficacy of DCCR treatment.

Conditions

Prader-Willi Syndrome

Keywords

PWS; Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (2)

• Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported

  Safety analyses will be conducted in all participants who receive at least one dose of DCCR. Adverse events will be described by type and level of severity.

  Baseline to end of OLE (up to 4 years)

• Change from RW Period Baseline in HQ-CT Total Score

  Hyperphagia-related behaviors will be assessed by the hyperphagia questionnaire for clinical trials (HQ-CT), an instrument designed to measure symptoms of food related preoccupations and behaviors. The HQ-CT consists of nine items with responses ranging from 0-4 (best to worst). Scores from 9 items will be summed for a possible total score range of 0-36.

  RW Period Baseline to Week 16

Secondary Outcomes (5)

• Change from Baseline in HQ-CT Total Score

  Baseline to end of OLE (up to 4 years)

• Change in Body Fat Mass

  Baseline to end of OLE (up to 4 years)

• Clinical Global Impression of Improvement (CGI-I)

  RW Period Week 16

• Clinical Global Impression of Severity (CGI-S)

  RW Period Week 16

• Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported

  through the end of the RW Period, 16 weeks

Study Arms (3)

OLE DCCR

EXPERIMENTAL

75 - 525 mg DCCR

Drug: DCCR

RW DCCR

EXPERIMENTAL

75 - 525 mg DCCR

Drug: DCCR

RW Placebo

PLACEBO COMPARATOR

75 - 525 mg Placebo for DCCR

Drug: Placebo for DCCR

Interventions

DCCR DRUG

Once daily oral administration of open-label DCCR tablet(s) during the OLE Period

OLE DCCR

Placebo for DCCR DRUG

Once daily oral administration of double-blind (placebo for DCCR) tablet(s) during the RW Period

RW 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:

• Successful completion of clinical study C601
• Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (subjects, as appropriate)

You may not qualify if:

• 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 90 days after study participation
• Any new disease, condition, or circumstance which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol (e.g., an anticipated change of care setting)
• Provide voluntary, written informed consent (parent\[s\] / legal guardian\[s\] of participant); provide voluntary, written assent (participants, as appropriate); this includes consent for randomization and potential treatment with placebo for up to 16 weeks
• Currently participating in clinical study C602 and complete the OLE End of Treatment Visit procedures
• Positive urine pregnancy test (in females of child-bearing potential)
• Females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation

Sponsors & Collaborators

• Soleno Therapeutics, Inc.: lead

Study Sites (28)

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, 37212, United States

Location

Research Institute of Dallas

Dallas, Texas, 75231, United States

Location

University of Utah

Salt Lake City, Utah, 84108, 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

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 (2)

• 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

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 Design

• Study Type: interventional
• Phase: phase 3
• 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: September 27, 2018
• First Posted: October 22, 2018
• Study Start: October 1, 2018
• Primary Completion: August 17, 2023
• Study Completion: August 17, 2023
• Last Updated: April 19, 2024

Record last verified: 2024-04

Locations

US (20); GB (8)