Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Beloranib) in Obese Subjects With Prader-Willi Syndrome (bestPWS)

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ClinicalTrials.gov Identifier: NCT02179151

Recruitment Status : Terminated (FDA Clinical Hold)

First Posted : July 1, 2014

Last Update Posted : January 26, 2017

Sponsor:

Information provided by (Responsible Party):

Zafgen, Inc.

Study Description

Brief Summary:

The purpose of this study is to evaluate efficacy and safety of ZGN-440 (beloranib) in obese adolescent and adult subjects with Prader-Willi Syndrome.

Condition or disease	Intervention/treatment	Phase
Prader-Willi Syndrome Obesity	Drug: ZGN-440 for Injectable Suspension Drug: ZGN-440 Placebo for Injectable Suspension	Phase 3

Detailed Description:

Phase 3, Randomized, Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Subcutaneous Beloranib in Suspension) in Obese Subjects with Prader-Willi Syndrome to Evaluate Total Body Weight, Food-related Behavior, and Safety Over 6 Months

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	108 participants
Allocation:	Randomized
Intervention Model:	Parallel Assignment
Masking:	Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:	Treatment
Official Title:	Randomized, Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Subcutaneous Beloranib in Suspension) in Obese Subjects With Prader-Willi Syndrome to Evaluate Total Body Weight, Food-related Behavior, and Safety Over 6 Months
Study Start Date :	September 2014
Actual Primary Completion Date :	December 2015
Actual Study Completion Date :	October 2016

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Prader-Willi syndrome

MedlinePlus related topics: Prader-Willi Syndrome

Genetic and Rare Diseases Information Center resources: Prader-Willi Syndrome

U.S. FDA Resources

Arms and Interventions

Arm	Intervention/treatment
Placebo Comparator: Placebo Intervention: ZGN-440 Placebo for Injectable Suspension	Drug: ZGN-440 Placebo for Injectable Suspension Subjects will receive placebo twice weekly subcutaneous injections for up to 28 weeks. Other Name: Placebo
Experimental: ZGN-440 Injectable Suspension (1.8 mg) Intervention: ZGN-440 for Injectable Suspension	Drug: ZGN-440 for Injectable Suspension Subjects will receive ZGN-440 twice weekly subcutaneous injections for up to 28 weeks. Other Names: ZGN-440 Beloranib
Experimental: ZGN-440 Injectable Suspension (2.4 mg) Intervention: ZGN-440 for Injectable Suspension	Drug: ZGN-440 for Injectable Suspension Subjects will receive ZGN-440 twice weekly subcutaneous injections for up to 28 weeks. Other Names: ZGN-440 Beloranib

Outcome Measures

Primary Outcome Measures :

Change in total body weight [ Time Frame: Baseline to Week 29 ]
Change in hyperphagia-related behavior as measured by total score of a Hyperphagia Questionnaire [ Time Frame: Baseline to Week 29 ]

Secondary Outcome Measures :

Change in LDL cholesterol [ Time Frame: Baseline to Week 29 ]
Change in HDL cholesterol [ Time Frame: Baseline to Week 29 ]
Change in total body mass as measured by DXA [ Time Frame: Baseline to Week 29 ]
Change in total body fat mass as measured by DXA [ Time Frame: Baseline to Week 29 ]
Change in triglyceride [ Time Frame: Baseline to Week 29 ]

Eligibility Criteria

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Ages Eligible for Study:	12 Years to 65 Years (Child, Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Confirmed genetic diagnosis of Prader-Willi Syndrome
Age 12-65
Obesity
Age 12-17: BMI ≥ 95th percentile for age and gender
Age 18-65: BMI ≥27 to ≤60 kg/m2

Exclusion Criteria:

Subjects living in a group home ≥ 50% of the time
Recent use (within 3 months) of weight loss agents including herbal medication
Poorly controlled severe psychiatric disorders

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02179151

Locations

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United States, California
University of California, Davis
Sacramento, California, United States, 95817
UCSD: Rady Children's Hospital
San Diego, California, United States, 92123
United States, Colorado
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
United States, Kansas
Kansas University Medical Center
Kansas City, Kansas, United States, 66160
United States, Maryland
National Institute of Child Health
Bethesda, Maryland, United States, 20892
United States, Massachusetts
Children's Hospital Boston
Boston, Massachusetts, United States, 02115
United States, Minnesota
Children's Hospital and Clinics of Minnesota
St. Paul, Minnesota, United States, 55102
United States, Missouri
Saint Louis University
St. Louis, Missouri, United States, 63104
United States, New York
Winthrop University
Mineola, New York, United States, 11501
United States, Ohio
University Hospitals Case Medical Center
Cleveland, Ohio, United States, 44106
United States, Tennessee
Vanderbilt University
Nashville, Tennessee, United States, 37235
United States, Texas
Texas Children's Hospital
Houston, Texas, United States, 77030
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84112
United States, Washington
Seattle Children's Research Institute
Seattle, Washington, United States, 98105

Sponsors and Collaborators

Zafgen, Inc.

Investigators

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Study Director:	Dennis Kim, MD	Zafgen, Inc.

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

McCandless SE, Yanovski JA, Miller J, Fu C, Bird LM, Salehi P, Chan CL, Stafford D, Abuzzahab MJ, Viskochil D, Barlow SE, Angulo M, Myers SE, Whitman BY, Styne D, Roof E, Dykens EM, Scheimann AO, Malloy J, Zhuang D, Taylor K, Hughes TE, Kim DD, Butler MG. Effects of MetAP2 inhibition on hyperphagia and body weight in Prader-Willi syndrome: A randomized, double-blind, placebo-controlled trial. Diabetes Obes Metab. 2017 Dec;19(12):1751-1761. doi: 10.1111/dom.13021. Epub 2017 Jul 13.

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Responsible Party:	Zafgen, Inc.
ClinicalTrials.gov Identifier:	NCT02179151
Other Study ID Numbers:	ZAF-311
First Posted:	July 1, 2014 Key Record Dates
Last Update Posted:	January 26, 2017
Last Verified:	January 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	No

Keywords provided by Zafgen, Inc.:


Prader-Willi Syndrome Obesity Hyperphagia

Additional relevant MeSH terms:

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Prader-Willi Syndrome Syndrome Disease Pathologic Processes Obesity Overnutrition Nutrition Disorders Intellectual Disability Neurobehavioral Manifestations Neurologic Manifestations Nervous System Diseases	Abnormalities, Multiple Congenital Abnormalities Chromosome Disorders Genetic Diseases, Inborn CKD732 Angiogenesis Inhibitors Angiogenesis Modulating Agents Growth Substances Physiological Effects of Drugs Growth Inhibitors Antineoplastic Agents

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