An Efficacy, Safety, and Pharmacokinetics Study of Beloranib in Obese Subjects With Prader-Willi Syndrome

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Obesity, Over-weight, Prader-Willi Syndrome

Intervention / Treatment
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* Drug: ZGN-440 sterile diluent
* Drug: 1.2 mg ZGN-440 for injectable suspension
* Drug: 1.8 mg ZGN-440 for injectable suspension


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 16 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: ZGN-440 sterile diluent<br>Subjects will receive placebo twice weekly subcutaneous injections for up to 6 weeks. | Drug: ZGN-440 sterile diluent<br>* ZGN-440 sterile diluent/placebo<br>* Other names: Placebo;|
| Experimental: 1.2 mg ZGN-440 for injectable suspension<br>Subjects will receive ZGN-440 for injectable suspension (beloranib) twice weekly subcutaneous injections for up to 8 weeks. | Drug: 1.2 mg ZGN-440 for injectable suspension<br>* 1.2 mg beloranib<br>* Other names: Beloranib;|
| Experimental: 1.8 mg ZGN-440 for injectable suspension<br>Subjects will receive ZGN-440 for injectable suspension (beloranib) twice weekly subcutaneous injections for up to 8 weeks. | Drug: 1.8 mg ZGN-440 for injectable suspension<br>* 1.8 mg beloranib<br>* Other names: Beloranib;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent change in body weight from baseline to the end of the randomized dosing period. |  | 4 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in body weight (kg) from baseline to the end of the randomized dosing period |  | 4 weeks | 
| 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 |  | 4 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Beloranib, ZGN-440, ZGN-440 for injectable suspension, ZGN-433