Mucopolysaccharidosis Type II

Study to Evaluate the Safety and Efficacy of HMI-203 (juMPStart Trial)

A Phase 1 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of HMI-203 in ERT-Treated Adults with Mucopolysaccharidosis Type II (MPS II) (juMPStart Trial)

About: This Phase 1 study will evaluate the safety and efficacy of HMI-203 gene therapy in adult male participants with MPS II currently being treated with standard-of-care idursulfase ERT or equivalent. Participants will receive a single dose of HMI-203 administered intravenously. There are 3 planned dose cohorts which will consist of 3 participants each.

Ages Eligible: 18 years to 30 years old

Sexes Eligible for Study: All 

Status: Recruitment starting soon.

For more information about this study, including eligibility criteria, please go to the study’s page. If you are interested in participating in this study or have questions about research, please contact Samantha Norman, Clinical Research Coordinator.