NCT05701774

Brief Summary

The purpose of this is study is to evaluate the long-term safety of DCCR (diazoxide choline) extended-release tablets) in patients with Prader-Willi syndrome.

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
83

participants targeted

Target at below P25 for phase_3

Timeline
24mo left

Started Jan 2023

Longer than P75 for phase_3

Geographic Reach
2 countries

22 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress63%
Jan 2023Jun 2028

First Submitted

Initial submission to the registry

January 18, 2023

Completed
9 days until next milestone

First Posted

Study publicly available on registry

January 27, 2023

Completed
4 days until next milestone

Study Start

First participant enrolled

January 31, 2023

Completed
5.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2028

Last Updated

April 2, 2025

Status Verified

March 1, 2025

Enrollment Period

5.3 years

First QC Date

January 18, 2023

Last Update Submit

March 28, 2025

Conditions

Outcome Measures

Primary Outcomes (1)

  • Safety of DCCR (adverse events. Safety analyses will be conducted in all subjects who receive at least one dose of DCCR in the C614 study. ( Adverse events)

    Assess the safety of DCCR by evaluating the incidence and severity of adverse events. Safety analyses will be conducted in all subjects who receive at least one dose of DCCR in the C614 study. Adverse events will be described by type and level of severity.

    Baseline to End of Study or until resolution of certain adverse events

Study Arms (1)

DCCR

EXPERIMENTAL

75 - 525 mg DCCR

Drug: DCCR

Interventions

DCCRDRUG

Once daily oral administration

DCCR

Eligibility Criteria

Age4 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (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 (subjects, as appropriate)
  • Participant must:
  • Have participated in and completed the Study C602 Randomized Withdrawal (RW) Period;
  • Have participated in but discontinued from the Study C602 RW Period and at least 16 weeks have elapsed since the date of their randomization into the C602 RW Period; or
  • Have participated in Study C602 OLE period, did not consent to participate in the RW Period, and at least 16 weeks have elapsed since the date of their C602 Open-Label Extension End of Treatment Visit.

You may not qualify if:

  • 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.
  • Participation in a clinical study of an investigational drug (including approved drugs for unapproved uses), investigational device, or therapeutic intervention subsequent to the C602 Open-Label Extension End of Treatment Visit.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (22)

UC Irvine

Orange, California, 92868, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

Rady Children's Hospital of San Diego

San Diego, California, 92123, United States

Location

U of Florida Gainesville

Gainesville, Florida, 32608, United States

Location

Emory Children's Center

Atlanta, Georgia, 30329, United States

Location

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

National Institutes of Health

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 Hospital and Clinic 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

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, 84112, United States

Location

Seattle Children's

Seattle, Washington, 98105, United States

Location

Fulbourn Hospital

Cambridge, CB21 5EF, United Kingdom

Location

The Queen Elizabeth University

Glasgow, G51 4TF, United Kingdom

Location

Hull and East Yorkshire Hospitals NHS Trust

Hull, HU3 2JZ, United Kingdom

Location

Royal London Hospital

London, E1 1BB, United Kingdom

Location

Chelsea and Westminster Hospital

London, SW10 9NH, United Kingdom

Location

MeSH Terms

Conditions

Prader-Willi Syndrome

Condition Hierarchy (Ancestors)

Intellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesChromosome DisordersGenetic Diseases, InbornImprinting DisordersObesityOverweightOvernutritionNutrition DisordersNutritional and Metabolic Diseases

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 18, 2023

First Posted

January 27, 2023

Study Start

January 31, 2023

Primary Completion (Estimated)

June 1, 2028

Study Completion (Estimated)

June 1, 2028

Last Updated

April 2, 2025

Record last verified: 2025-03

Data Sharing

IPD Sharing
Will not share

Locations