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

No Placebo-Only Group

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline; weeks -1, 1, 4, 8, 12, 24, 52, 78, 104, and 260

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

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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

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