DCCR for the Treatment of Hyperphagia in PWS

Study Purpose

Soleno Therapuetics is conducting a Phase 3 study of DCCR to measure the drugs efficacy for treating hyperphagia in PWS. The study seeks to enroll 105 patients with PWS ages 4 years and older. The study will be a 15 week, randomized, double-blind, placebo-controlled study of Diazoxide Choline Controlled-Release Tablet (DCCR) and will require a once daily oral administration. The initial 15 week study will be followed by a 9-month extension during which time all patients will receive DCCR. 

Protocol Update: A recent amendment to the study protocol now allows patients between 20kg and 130kg to enroll in the study, provided they meet all eligibility criteria. Individual study sites will be able to enroll patients in the new weight band (20-30kg) once the appropriate tablet strengths come available, which will likely be in the September timeframe. Patients who are interested in enrolling should contact their nearest study coordinator.

A webinar sharing details on this study was recorded February, 2019. You can watch the webinar below or read a transcription of the webinar on our blog

 

Recruitment Criteria

Study Type: Randomized, double-blind, placebo-controlled
Eligible Ages: 4 years and older and at least 20kg
Other Criteria:

Trial Details

Trial IdNCT03440814
Phase: 3
Duration: 15 weeks, followed by optional 9 month open label extension
Status:
 recruiting
Lead Sponsor: Soleno
Countries: United States
Additional Details: 

Eligibility Criteria

Inclusion Criteria:

  • Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (patients, as appropriate)
  • Genetically-confirmed Prader-Willi syndrome
  • 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

Exclusion Criteria:

  • 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

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you. More information on this study is available on the study information page at clincaltrials.gov.

This Study is enrolling at the following sites:

United States, California

University of California, Irvine
Orange, California
Contact: Margaret Knight    949-824-0521    mnrobins@uci.edu 
Principal Investigator: Virginia Kimonis, MD 

Stanford University
Palo Alto, California
Principal Investigator: David Stevenson, MD
Contact: Alicia Harnett    650-723-0441    aharnett@stanford.edu   

Rady Children's Hospital
San Diego, California
Principal Investigator: Lynne M. Bird, MD 
Contact: Rachel Winograd, R.N.    858-966-8453    Rwinograd@rchsd.org 

United States, Colorado

Children's Hospital Colorado
Aurora, Colorado
Principal Investigator: Shawn McCandless, MD     
Contact: Marcie Terry    720-777-8439    Marcelle.Terry@childrenscolorado.org

United States, Florida

University of Florida Gainesville
Gainesville, Florida
Principal Investigator: Jennifer L Miller, MD
Contact: Beverly P Giordano, MS, PNP 352-294-5280  bgiordano@ufl.edu

United States, Georgia

Emory Children's Center
Atlanta, Georgia, United States, 30322
Principal Investigator: Eric Felner, MD  
Contact: Margaret (Peggy) Jenkins    404-712-0051    mjenk03@emory.edu   
       

United States, Kansas

Kansas University Medical Center
Kansas City, Kansas
Principal Investigator: Merlin Butler, MD
Contact: Yolanda Harness    913-574-0302    yharness@kumc.edu   

United States, Maryland

National Institutes of Health
Bethesda, Maryland
Principal Investigator: Jack Yanovski, MD, Ph.D
Contact: Elisabeth Davis, BA    301-496-6726    elisabeth.davis2@nih.gov
Contact: Shelia Brady, MSN    301-451-3783    bradys@mail.nih.gov  

United States, Massachusetts

Boston Children's Hospital
Boston, Massachusetts
Principal Investigator: Amy Fleischman, MD
Contact: Andrea Hale, RN, BSN, MPH, CCRP    617-918-2867    andrea.hale@childrens.harvard.edu 

United States, Michigan

Sparrow Clinical Research Institute
Lansing, Michigan
Principal Investigator: Elizabeth Littlejohn, MD   
Contact: Jennifer Boak    517-364-5733    jennifer.boak@sparrow.org   


United States, Minnesota

Children's Minnesota
Saint Paul, Minnesota
Principal Investigator: Jennifer Abuzzahab, MD
Contact: Brittany Machus, MA, CCRP    651-220-5730    brittany.machus@childrensmn.org 

United States, New York

NYU Winthrop Hospital
Mineola, New York
Principal Investigator: Moris Angulo, MD
Contact: Marilyn Richardson    516-663-9582    marilyn.richardson@nyulangone.org 

United States, Ohio

University Hospitals Cleveland Medical Center
Cleveland, Ohio
Principal Investigator: Lori-Anne Schillaci, MD, FAAP
Contact: Audrey Lynn, Ph.D    216-844-7124    audrey.lynn@uhhospitals.org

The Research Institute at Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Principal Investigator: Kathryn Obrynba, MD
Contact: Emily Viall    614-722-2630    Emily.Viall@nationwidechildrens.org

United States, Tennessee

Vanderbilt University
Nashville, Tennessee
Principal Investigator: Ashley Hall Shoemaker, MD
Contact: Sarah N McKeel, R.N., BSN    615-875-3772    sarah.n.mckeel@vumc.org

United States, Utah

University of Utah
Salt Lake City, Utah, United States, 84113
Principal Investigator: David Viskochil, MD   
Contact: Carrie Bailey    801-587-3605    Carrie.Bailey@hsc.utah.edu   

United States, Washington

Seattle Children's Hospital
Seattle, Washington
Principal Investigator: Parisa Salehi, MD
Contact: Sue Kearns, BMS 206-987-2640 sue.kearns@seattlechildrens.org

United Kingdom

Birmingham Women's and Children's Hospital
Birmingham, United Kingdom, B4 6NH
Principal Investigator: Timothy Barrett   
Contact: Josie Goodby       josie.goodby@nhs.net   

Royal London Hospital
London, United Kingdom, E1 1BB
Principal Investigator: Evelien Gevers
Contact: Tara Murray    020 5941455    tara.murray@bartshealth.nhs.uk   

Chelsea and Westminster Hospital
London, United Kingdom, SW10 9NH
Principal Investigator: Nicola Bridges  
Contact: Rhian Bull       Rhian.Bull@chelwest.nhs.uk   
    

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