Status:
WITHDRAWN
Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II
Lead Sponsor:
Homology Medicines, Inc
Conditions:
Mucopolysaccharidosis II
Eligibility:
MALE
18-45 years
Phase:
PHASE1
Brief Summary
Phase 1, open-label, sequential ascending dose-escalation study. Designed to evaluate the safety and efficacy of a single IV infusion of investigational gene therapy HMI-203. Males, ages 18 to 45 year...
Detailed Description
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. Par...
Eligibility Criteria
Inclusion
- Key
- Adult males 18-45 years of age at the time of informed consent
- 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
- Kaufman Brief Intelligence Test-Second Edition (KBIT2) score ≥ 80
- Compliance with regular treatments of ERT for MPS II for at least 12 months prior to enrollment
- Clinically stable relative to urinary GAG levels, ambulation, and cardiopulmonary status for 12 months preceding enrollment
- Key
Exclusion
- Multiple sulfatase disorder as determined by abnormal activity of another lysosomal sulfatase
- Unresponsive and/or intolerant to idursulfase treatment
- History of BMT, stem cell transplant, or gene therapy
- Presence of anti-capsid neutralizing antibodies
- ALT or AST \> ULN; Total or Direct bilirubin \> ULN
- International normalized ratio (INR) \>1.2 ULN
- Hematology values below the normal range
- Hemoglobin A1c ≥ 6.5% or fasting glucose ≥126 mg/dL
- Contraindication to corticosteroid or tacrolimus use
- Any condition that would not allow the potential participant to complete follow-up examinations during the course of the study or, in the opinion of the investigator, makes the potential participant unsuitable for the study
Key Trial Info
Start Date :
September 8 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2029
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT05238324
Start Date
September 8 2022
End Date
January 1 2029
Last Update
August 28 2023
Active Locations (6)
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1
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
2
Yale Center for Clinical Investigation
New Haven, Connecticut, United States, 06519
3
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
4
University of Utah Pediatric Genetic & Metabolism Clinic
Salt Lake City, Utah, United States, 84113