NCT03422627

Safety and Efficacy of Efavaleukin Alfa in Subjects With Steroid Refractory Chronic Graft Versus Host Disease

A Phase 1b/2 Open-label Study Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Efavaleukin Alfa in Adult Subjects With Steroid Refractory Chronic Graft Versus Host Disease

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: Amgen · Industry
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

Phase 1b: To evaluate the safety and tolerability of multiple ascending doses of efavaleukin alfa in subjects with steroid refractory chronic graft versus host disease (cGVHD). Phase 2: To evaluate the efficacy of efavaleukin alfa in subjects with steroid refractory cGVHD as measured by overall response rate (ORR) at 16 weeks according to the 2014 cGVHD NIH Consensus Criteria. Due to early termination, the Phase 2 portion of this study was not conducted.

Conditions

• Chronic Graft Versus Host Disease cGVHD

Interventions

Type	Name	Description
DRUG	Efavaleukin Alfa	Phase 1: This study will be conducted as a multiple ascending dose (MAD) study. Each dosing cohort will consist of between 3 and 6 subjects who will receive efavaleukin alfa subcutaneously (SC) either every week or every 2 weeks plus protocol permitted background therapy for 52 weeks. At the discretion of the Sponsor, following discussion and agreement between the principal investigator and medical monitor, subjects responding to efavaleukin alfa (as assessed by the end of week 50), who wish to continue treatment, may continue to receive efavaleukin alfa treatment at their current dosing regimen for up to an additional 208 weeks through an extended dosing period. All subjects who continue to receive efavaleukin alfa during the extended dosing period will be reevaluated every 6 months for their response to treatment. Phase 2: All subjects will receive the recommended phase 2 dose (RP2D) of efavaleukin alfa for 52 weeks.

Timeline

• Start date: 2018-04-27
• Primary completion: 2022-10-13
• Completion: 2022-10-13
• First posted: 2018-02-06
• Last updated: 2023-11-22
• Results posted: 2023-11-22

Locations

14 sites across 4 countries: United States, Belgium, France, Japan

Regulatory

• FDA-regulated drug study