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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

Study Summary

This trial is designed to see if a new gene therapy for Hunter syndrome is safe and works. Males ages 18-45 who are currently taking idursulfase ERT (or the equivalent) can participate. The trial will last for 5 years.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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 outcome measures

Change in CSF levels I2S activity and concentration

Change in CSF levels of dermatan sulfate

Change in CSF levels of heparan sulfate

+16 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

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Homology Medicines, IncLead Sponsor

5 Previous Clinical Trials

12,041 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are any additional participants able to enter this clinical trial?

"The research, which was initially made public on December 1st 2022, is currently recruiting participants according to the clinicaltrials.gov database and has last been edited November 14th 2022."

Answered by AI

Has the FDA sanctioned Genetic HMI-203 for public use?

"Due to the limited data avaiable, our team rated Genetic HMI-203's safety as 1 on a scale of one to three. This is because it is currently in Phase 1 trials and has not been fully tested for efficacy or safety yet."

Answered by AI

What qualifications must a patient meet to be eligible for this trial?

"Nine MPS II patients, aged 18-45 years of age and with a KBIT2 score ≥ 80 are being recruited to join this clinical trial. They must be compliant with ERT treatments for 12 months prior to enrolment; have been clinically stable relative to urinary GAG levels, ambulation and cardiopulmonary status over the last year; possess the mental capacity necessary for understanding the study’s purpose and risks; demonstrate their ability to comply with all procedures during 5 years after gene therapy administration, as well as execute 6MWT successfully."

Answered by AI

Is the patient pool for this experiment limited to individuals younger than 36?

"Individuals aged 18-45 years old are eligible for this medical trial."

Answered by AI

What is the total count of participants for this experiment?

"Affirmative. Clinicaltrials.gov corroborates that the trial, which was initially listed on December 1st 2022, is still open to new patients. Only 9 participants are needed at 3 distinct sites for this study."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2022. "Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05238324.