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

Primary Purpose

Prader-Willi Syndrome, Obesity

Status
Terminated
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
ZGN-440 for Injectable Suspension
ZGN-440 Placebo for Injectable Suspension
Sponsored by
Zafgen, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Prader-Willi Syndrome focused on measuring Prader-Willi Syndrome, Obesity, Hyperphagia

Eligibility Criteria

12 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of California, Davis
  • UCSD: Rady Children's Hospital
  • Children's Hospital of Colorado
  • University of Florida
  • Kansas University Medical Center
  • National Institute of Child Health
  • Children's Hospital Boston
  • Children's Hospital and Clinics of Minnesota
  • Saint Louis University
  • Winthrop University
  • University Hospitals Case Medical Center
  • Vanderbilt University
  • Texas Children's Hospital
  • University of Utah
  • Seattle Children's Research Institute

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Placebo Comparator

Experimental

Experimental

Arm Label

Placebo

ZGN-440 Injectable Suspension (1.8 mg)

ZGN-440 Injectable Suspension (2.4 mg)

Arm Description

Intervention: ZGN-440 Placebo for Injectable Suspension

Intervention: ZGN-440 for Injectable Suspension

Intervention: ZGN-440 for Injectable Suspension

Outcomes

Primary Outcome Measures

Change in total body weight
Change in hyperphagia-related behavior as measured by total score of a Hyperphagia Questionnaire

Secondary Outcome Measures

Change in LDL cholesterol
Change in HDL cholesterol
Change in total body mass as measured by DXA
Change in total body fat mass as measured by DXA
Change in triglyceride

Full Information

First Posted
June 25, 2014
Last Updated
January 24, 2017
Sponsor
Zafgen, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT02179151
Brief Title
Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Beloranib) in Obese Subjects With Prader-Willi Syndrome
Acronym
bestPWS
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 Type
Interventional

2. Study Status

Record Verification Date
January 2017
Overall Recruitment Status
Terminated
Why Stopped
FDA Clinical Hold
Study Start Date
September 2014 (undefined)
Primary Completion Date
December 2015 (Actual)
Study Completion Date
October 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Zafgen, Inc.

4. Oversight

Data Monitoring Committee
Yes

5. 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.
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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome, Obesity
Keywords
Prader-Willi Syndrome, Obesity, Hyperphagia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
108 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Intervention: ZGN-440 Placebo for Injectable Suspension
Arm Title
ZGN-440 Injectable Suspension (1.8 mg)
Arm Type
Experimental
Arm Description
Intervention: ZGN-440 for Injectable Suspension
Arm Title
ZGN-440 Injectable Suspension (2.4 mg)
Arm Type
Experimental
Arm Description
Intervention: ZGN-440 for Injectable Suspension
Intervention Type
Drug
Intervention Name(s)
ZGN-440 for Injectable Suspension
Other Intervention Name(s)
ZGN-440, Beloranib
Intervention Description
Subjects will receive ZGN-440 twice weekly subcutaneous injections for up to 28 weeks.
Intervention Type
Drug
Intervention Name(s)
ZGN-440 Placebo for Injectable Suspension
Other Intervention Name(s)
Placebo
Intervention Description
Subjects will receive placebo twice weekly subcutaneous injections for up to 28 weeks.
Primary Outcome Measure Information:
Title
Change in total body weight
Time Frame
Baseline to Week 29
Title
Change in hyperphagia-related behavior as measured by total score of a Hyperphagia Questionnaire
Time Frame
Baseline to Week 29
Secondary Outcome Measure Information:
Title
Change in LDL cholesterol
Time Frame
Baseline to Week 29
Title
Change in HDL cholesterol
Time Frame
Baseline to Week 29
Title
Change in total body mass as measured by DXA
Time Frame
Baseline to Week 29
Title
Change in total body fat mass as measured by DXA
Time Frame
Baseline to Week 29
Title
Change in triglyceride
Time Frame
Baseline to Week 29

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dennis Kim, MD
Organizational Affiliation
Zafgen, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
University of California, Davis
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
UCSD: Rady Children's Hospital
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Children's Hospital of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Kansas University Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
National Institute of Child Health
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Facility Name
Children's Hospital Boston
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Children's Hospital and Clinics of Minnesota
City
St. Paul
State/Province
Minnesota
ZIP/Postal Code
55102
Country
United States
Facility Name
Saint Louis University
City
St. Louis
State/Province
Missouri
ZIP/Postal Code
63104
Country
United States
Facility Name
Winthrop University
City
Mineola
State/Province
New York
ZIP/Postal Code
11501
Country
United States
Facility Name
University Hospitals Case Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Vanderbilt University
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37235
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
Seattle Children's Research Institute
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
28556449
Citation
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.
Results Reference
derived

Learn more about this trial

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

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