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

Withdrawn before enrolling · Phase 1

Conditions studied: Mucopolysaccharidosis II

In brief

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.

Key facts

Study ID

NCT05238324

Run by

Homology Medicines, Inc

People needed

0

Starts

2022-09-08

Expected to finish

2029-01-01

Last updated by the study team

2023-08-28

Who can join

Age: 18 and older, up to 45. Sex: male. Healthy volunteers: not accepted.

Where it is running

• UCSF Benioff Children's Hospital Oakland — Oakland, California, United States
• Yale Center for Clinical Investigation — New Haven, Connecticut, United States
• Hackensack University Medical Center — Hackensack, New Jersey, United States
• University of Utah Pediatric Genetic & Metabolism Clinic — Salt Lake City, Utah, United States
• Lysosomal and Rare Disorders Research and Treatment Center, Inc. — Fairfax, Virginia, United States
• M.A.G.I.C. Clinic, Ltd. — Calgary, Alberta, Canada

Full record on ClinicalTrials.gov

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