Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Summary

Phase 1, open-label, sequential ascending dose-escalation study. Designed to evaluate the safety and efficacy of a single IV infusion of investigational gene therapy HMI-203. Males, ages 18 to 45 years inclusive, with MPS II (Hunter syndrome) currently receiving idursulfase ERT (or the equivalent) are eligible to participate. Participants will be followed for safety and efficacy for 5 years.

Timeline

Start

2022-09-08

Primary completion

2024-10

Completion

2029-01

Outcome

Outcome not reported

Stopped: “Homology Medicines has discontinued development of this program.”

Drugs

Indications