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

Phase 3 Terminated 108 enrolled Zafgen, Inc.

RandomizedParallel-groupQuadruple-blindTreatment

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.

Timeline

Start: 2014-09
Primary completion: 2015-12
Completion: 2016-10

Outcome

Met primary endpoint

Stopped: “FDA Clinical Hold”

paper Improvement (reduction) in HQ-CT total score was greater in the 1.8 mg (mean difference -6.3, 95% CI -9.6 to -3.0; P = .0003) and 2.4 mg PMID 28556449 ↗

paper Compared with placebo, weight change was greater with 1.8 mg (mean difference - 8.2%, 95% CI -10.8 to -5.6; P < .0001) and 2.4 mg beloranib (-9.5%, 95% CI -12.1 to -6.8; P < .0001). PMID 28556449 ↗

Publications

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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Beloranib	Small molecule	1.8 mg	Subcutaneous
Subject	Beloranib	Small molecule	2.4 mg	Subcutaneous