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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Zafgen, Inc.
ClinicalTrials.gov Identifier:
NCT01818921
First received: March 21, 2013
Last updated: December 17, 2013
Last verified: December 2013
  Purpose

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.


Condition Intervention Phase
Obesity
Over-weight
Prader-Willi Syndrome
Drug: ZGN-440 sterile diluent
Drug: 1.2 mg ZGN-440 for injectable suspension
Drug: 1.8 mg ZGN-440 for injectable suspension
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Zafgen, Inc.:

Primary Outcome Measures:
  • Percent change in body weight from baseline to the end of the randomized dosing period. [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in body weight (kg) from baseline to the end of the randomized dosing period [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]

Enrollment: 17
Study Start Date: June 2013
Study Completion Date: November 2013
Primary Completion Date: November 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: ZGN-440 sterile diluent
Subjects will receive placebo twice weekly subcutaneous injections for up to 6 weeks.
Drug: ZGN-440 sterile diluent
ZGN-440 sterile diluent/placebo
Other Names:
  • ZGN-440 sterile diluent
  • Placebo
Experimental: 1.2 mg ZGN-440 for injectable suspension
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
1.2 mg beloranib
Other Names:
  • ZGN-440 for injectable suspension
  • ZGN-440
  • ZGN-433
  • Beloranib
Experimental: 1.8 mg ZGN-440 for injectable suspension
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
1.8 mg beloranib
Other Names:
  • ZGN-440 for injectable suspension
  • ZGN-440
  • ZGN-433
  • Beloranib

  Eligibility

Ages Eligible for Study:   16 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01818921

Locations
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610-0296
Sponsors and Collaborators
Zafgen, Inc.
Investigators
Principal Investigator: Jennifer Miller, MD University of Florida
  More Information

No publications provided

Responsible Party: Zafgen, Inc.
ClinicalTrials.gov Identifier: NCT01818921     History of Changes
Other Study ID Numbers: ZAF-211
Study First Received: March 21, 2013
Last Updated: December 17, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Zafgen, Inc.:
Beloranib
ZGN-440
ZGN-440 for injectable suspension
ZGN-433

Additional relevant MeSH terms:
Prader-Willi Syndrome
Overweight
Syndrome
Abnormalities, Multiple
Body Weight
Chromosome Disorders
Congenital Abnormalities
Disease
Genetic Diseases, Inborn
Intellectual Disability
Nervous System Diseases
Neurobehavioral Manifestations
Neurologic Manifestations
Nutrition Disorders
Obesity
Overnutrition
Pathologic Processes
Signs and Symptoms

ClinicalTrials.gov processed this record on October 21, 2014