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

Recruiting at 5 trial locations

Overseen By Tracy McGregor, M.D.

Age: 18 - 65

Sex: Male

Trial Phase: Phase 1

Sponsor: Homology Medicines, Inc

Must be taking: Idursulfase ERT

Must not be taking: Corticosteroids, Tacrolimus

Disqualifiers: Multiple sulfatase disorder, BMT, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

Trial Summary

What is the purpose of this trial?

This trial tests a new gene therapy called HMI-203 in adult males with Hunter syndrome who are already on standard treatment. The therapy is given as a single IV infusion to introduce new genes that may help improve their condition.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it requires participants to be on regular enzyme replacement therapy (ERT) for MPS II. It's best to discuss your specific medications with the trial team.

Eligibility Criteria

Inclusion Criteria

You have been in good health for the past year, with no significant problems with urination, walking, or heart and lung function.

Has capacity and is able to understand the purpose and risks of the study and is willing, able and committed to comply with all study procedures for the duration of the trial (a total of 5 years after gene therapy administration)

Diagnosis of MPS II based on historically decreased I2S enzyme activity and elevated urine GAGs and/or presence of hemizygous IDS pathogenic variant

Exclusion Criteria

Presence of anti-capsid neutralizing antibodies

ALT or AST > ULN; Total or Direct bilirubin > ULN

You have undergone a bone marrow transplant, stem cell transplant, or gene therapy in the past.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single IV infusion of HMI-203 gene therapy

1 day

1 visit (in-person)

Dose Escalation

Participants are enrolled in one of three dose cohorts with intervals for safety and efficacy review

60 days for first cohort, 21 days for subsequent cohorts

Follow-up

Participants are monitored for safety and efficacy after treatment

5 years

Most frequent visits occur in the first year

Treatment Details

Interventions

• HMI-203

Participant Groups

Group I: HMI-203 Low Dose Level Cohort 1 Experimental Treatment 1 Intervention

Group II: HMI-203 Intermediate Dose Level Cohort 2 Experimental Treatment 1 Intervention

Group III: HMI-203 High Dose Level Cohort 3 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Homology Medicines, Inc

Lead Sponsor

Trials

6

Recruited

12,000+