Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS

Phase 3 Completed 33 enrolled Pfizer

Non-randomizedSingle-groupOpen-labelTreatment

Summary

This is a multicenter, open label, multi cohort study to evaluate the efficacy and safety of somatropin in a cohort of Japanese participants with PWS.

Timeline

Start: 2021-02-09
Primary completion: 2022-12-06
Completion: 2024-04-15

Outcome

Met primary endpoint

paper The study met its primary endpoint in the adult cohort PMID 40436776 ↗

Publications

Kawai M, Murakami N, Horikawa R, Muroya K, Fujisawa Y, Hoshino Y, Okayama A, Sato T, Ebata N, Ogata T. Improvement in body composition of Japanese participants with Prader-Willi syndrome following somatropin treatment: an open-label, multi cohort Phase 3 study. Endocr J. 2025 Aug 1;72(8):925-935. doi: 10.1507/endocrj.EJ24-0659. Epub 2025 May 28.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Somatropin	Protein / enzyme biologic	0.084 mg/kg	—
Subject	Somatropin	Protein / enzyme biologic	0.245 mg/kg	—