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

NCT ID: NCT03714373

Last Updated: 2024-04-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

115 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-10-01

Study Completion Date

2023-08-17

Brief Summary

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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.

Detailed Description

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115 PWS participants who completed clinical study C601 will be enrolled into the OLE Period. All participants in the Open Label Extension (OLE) Period will receive open-label DCCR. The actual number of participants eligible to enroll in the double-blind, placebo-controlled, randomized withdrawal (RW) period will be limited to those participants taking DCCR in the OLE Period at the time of the RW Period Visit 1 (Baseline/Randomization Visit).The treatment groups in the C602 RW Period are those participants randomized to receive DCCR and those participants randomized to receive Placebo. Participants will be randomized in a 1:1 ratio (DCCR:Placebo).

Conditions

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Prader-Willi Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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OLE DCCR

75 - 525 mg DCCR

Group Type EXPERIMENTAL

DCCR

Intervention Type DRUG

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

RW DCCR

75 - 525 mg DCCR

Group Type EXPERIMENTAL

DCCR

Intervention Type DRUG

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

RW Placebo

75 - 525 mg Placebo for DCCR

Group Type PLACEBO_COMPARATOR

Placebo for DCCR

Intervention Type DRUG

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

Interventions

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DCCR

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

Intervention Type DRUG

DCCR

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

Intervention Type DRUG

Placebo for DCCR

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

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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)


* 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

Exclusion Criteria

* 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)


* 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
Minimum Eligible Age

4 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Soleno Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of California, Irvine

Orange, California, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

Rady Children's Hospital San Diego

San Diego, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

University of Florida Gainesville

Gainesville, Florida, United States

Site Status

Emory Children's Center

Atlanta, Georgia, United States

Site Status

Indiana University School of Medicine

Indianapolis, Indiana, United States

Site Status

Kansas University Medical Center

Kansas City, Kansas, United States

Site Status

National Institutes of Health Hatfield Clinical Research Center

Bethesda, Maryland, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Sparrow Clinical Research Institute

Lansing, Michigan, United States

Site Status

Children's Minnesota

Saint Paul, Minnesota, United States

Site Status

St. Joseph's University Medical Center

Paterson, New Jersey, United States

Site Status

NYU Winthrop Hospital

Mineola, New York, United States

Site Status

University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Site Status

The Research Institute at Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Vanderbilt University

Nashville, Tennessee, United States

Site Status

Research Institute of Dallas

Dallas, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

The Queen Elizabeth University

Glasgow, Scottland, United Kingdom

Site Status

Hull and East Yorkshire Hospitals NHS Trust

Hull, Yorkshire, United Kingdom

Site Status

Birmingham Women's and Children's Hospital

Birmingham, , United Kingdom

Site Status

Fulbourn Hospital

Cambridge, , United Kingdom

Site Status

Aintree University Hospital NHS Foundation Trust

Liverpool, , United Kingdom

Site Status

Royal London Hospital

London, , United Kingdom

Site Status

Chelsea and Westminster Hospital

London, , United Kingdom

Site Status

Hammersmith Hospital

London, , United Kingdom

Site Status

Countries

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United States United Kingdom

References

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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.

Reference Type DERIVED
PMID: 38671361 (View on PubMed)

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.

Reference Type DERIVED
PMID: 37919617 (View on PubMed)

Other Identifiers

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C602

Identifier Type: -

Identifier Source: org_study_id

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