An Efficacy, Safety, and Pharmacokinetics Study of Beloranib in Obese Subjects With Prader-Willi Syndrome
Primary Purpose
Obesity, Over-weight, Prader-Willi Syndrome
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
ZGN-440 sterile diluent
1.2 mg ZGN-440 for injectable suspension
1.8 mg ZGN-440 for injectable suspension
Sponsored by
About this trial
This is an interventional treatment trial for Obesity focused on measuring Beloranib, ZGN-440, ZGN-440 for injectable suspension, ZGN-433
Eligibility Criteria
Inclusion Criteria:
- Confirmed diagnosis of Prader-Willi Syndrome due to chromosome 15 micro-deletion, maternal uniparental disomy, or imprinting defect
- BMI ≥25 kg/m2
- Type 2 diabetes mellitus is allowed
- Subject must agree to stay at the group home or under supervision of the group home or site staff (i.e. no home visits) for the duration of the study
- Stable body weight during the past 3 months, except for during home visits
Exclusion Criteria:
- Use of weight loss agents in the past 3 months
- Type 1 diabetes mellitus
- Current or anticipated chronic use of narcotics or opiates
Sites / Locations
- University of Florida
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Placebo Comparator
Experimental
Experimental
Arm Label
ZGN-440 sterile diluent
1.2 mg ZGN-440 for injectable suspension
1.8 mg ZGN-440 for injectable suspension
Arm Description
Subjects will receive placebo twice weekly subcutaneous injections for up to 6 weeks.
Subjects will receive ZGN-440 for injectable suspension (beloranib) twice weekly subcutaneous injections for up to 8 weeks.
Subjects will receive ZGN-440 for injectable suspension (beloranib) twice weekly subcutaneous injections for up to 8 weeks.
Outcomes
Primary Outcome Measures
Percent change in body weight from baseline to the end of the randomized dosing period.
Secondary Outcome Measures
Change in body weight (kg) from baseline to the end of the randomized dosing period
Change in hyperphagia behavior, drive, and severity score (total score) from baseline to the end of the randomized dosing period using the PWS Hyperphagia Questionnaire
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01818921
Brief Title
An Efficacy, Safety, and Pharmacokinetics Study of Beloranib in Obese Subjects With Prader-Willi Syndrome
Official Title
Randomized, Double-Blind, Placebo Controlled, Parallel Dose Ranging Phase 2a Trial of ZGN-440 (Subcutaneous Beloranib in Suspension), A Novel Methionine Aminopeptidase 2 Inhibitor, in Over-weight and Obese Subjects With Prader-Willi Syndrome to Evaluate Weight Reduction, Food-related Behavior, Safety, and Pharmacokinetics Over 4 Weeks Followed by Optional 4-Week Open-Label Extension
Study Type
Interventional
2. Study Status
Record Verification Date
July 2016
Overall Recruitment Status
Completed
Study Start Date
June 2013 (undefined)
Primary Completion Date
November 2013 (Actual)
Study Completion Date
November 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Zafgen, Inc.
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics for certain doses of beloranib in obese subjects with Prader-Willi Syndrome.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Obesity, Over-weight, Prader-Willi Syndrome
Keywords
Beloranib, ZGN-440, ZGN-440 for injectable suspension, ZGN-433
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
17 (Actual)
8. Arms, Groups, and Interventions
Arm Title
ZGN-440 sterile diluent
Arm Type
Placebo Comparator
Arm Description
Subjects will receive placebo twice weekly subcutaneous injections for up to 6 weeks.
Arm Title
1.2 mg ZGN-440 for injectable suspension
Arm Type
Experimental
Arm Description
Subjects will receive ZGN-440 for injectable suspension (beloranib) twice weekly subcutaneous injections for up to 8 weeks.
Arm Title
1.8 mg ZGN-440 for injectable suspension
Arm Type
Experimental
Arm Description
Subjects will receive ZGN-440 for injectable suspension (beloranib) twice weekly subcutaneous injections for up to 8 weeks.
Intervention Type
Drug
Intervention Name(s)
ZGN-440 sterile diluent
Other Intervention Name(s)
Placebo
Intervention Description
ZGN-440 sterile diluent/placebo
Intervention Type
Drug
Intervention Name(s)
1.2 mg ZGN-440 for injectable suspension
Other Intervention Name(s)
ZGN-440 for injectable suspension, ZGN-440, ZGN-433, Beloranib
Intervention Description
1.2 mg beloranib
Intervention Type
Drug
Intervention Name(s)
1.8 mg ZGN-440 for injectable suspension
Other Intervention Name(s)
ZGN-440 for injectable suspension, ZGN-440, ZGN-433, Beloranib
Intervention Description
1.8 mg beloranib
Primary Outcome Measure Information:
Title
Percent change in body weight from baseline to the end of the randomized dosing period.
Time Frame
4 weeks
Secondary Outcome Measure Information:
Title
Change in body weight (kg) from baseline to the end of the randomized dosing period
Time Frame
4 weeks
Title
Change in hyperphagia behavior, drive, and severity score (total score) from baseline to the end of the randomized dosing period using the PWS Hyperphagia Questionnaire
Time Frame
4 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Confirmed diagnosis of Prader-Willi Syndrome due to chromosome 15 micro-deletion, maternal uniparental disomy, or imprinting defect
BMI ≥25 kg/m2
Type 2 diabetes mellitus is allowed
Subject must agree to stay at the group home or under supervision of the group home or site staff (i.e. no home visits) for the duration of the study
Stable body weight during the past 3 months, except for during home visits
Exclusion Criteria:
Use of weight loss agents in the past 3 months
Type 1 diabetes mellitus
Current or anticipated chronic use of narcotics or opiates
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jennifer Miller, MD
Organizational Affiliation
University of Florida
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610-0296
Country
United States
12. IPD Sharing Statement
Learn more about this trial
An Efficacy, Safety, and Pharmacokinetics Study of Beloranib in Obese Subjects With Prader-Willi Syndrome
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