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Gene Therapy

Genetic HMI-203 for Hunter Syndrome

Phase 1
Waitlist Available
Research Sponsored by Homology Medicines, Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline; weeks -1, 1, 4, 8, 12, 24, 52, 78, 104, and 260
Awards & highlights

Summary

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.

Eligible Conditions
  • Hunter Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline; weeks -1, 1, 4, 8, 12, 24, 52, 78, 104, and 260
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline; weeks -1, 1, 4, 8, 12, 24, 52, 78, 104, and 260 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Evaluate the effect of HMI-203 single administration on plasma I2S activity within each dose cohort
Evaluate the effect of HMI-203 single administration on urinary GAG levels within each dose cohort
Evaluate the incidence and severity of adverse events of special interest (AESIs) after a single dose administration of HMI-203 (at each dose level) in adult participants with MPS II
+1 more
Secondary study objectives
Change in CSF levels I2S activity and concentration
Change in CSF levels of dermatan sulfate
Change in CSF levels of heparan sulfate
+8 more

Trial Design

3Treatment groups
Experimental Treatment
Group I: HMI-203 Low Dose Level Cohort 1Experimental Treatment1 Intervention
Group II: HMI-203 Intermediate Dose Level Cohort 2Experimental Treatment1 Intervention
Group III: HMI-203 High Dose Level Cohort 3Experimental Treatment1 Intervention

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Who is running the clinical trial?

Homology Medicines, IncLead Sponsor
5 Previous Clinical Trials
12,041 Total Patients Enrolled
~0 spots leftby Oct 2024
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