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

NCT05238324 · Status: WITHDRAWN · Phase: PHASE1 · Type: INTERVENTIONAL

Last updated 2023-08-28

No results posted yet for this study

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 years inclusive, with MPS II (Hunter syndrome) currently receiving idursulfase ERT (or the equivalent) are eligible to participate. Participants will be followed for safety and efficacy for 5 years.

Conditions

  • Mucopolysaccharidosis II

Interventions

BIOLOGICAL

Genetic HMI-203

HMI-203 delivered intravenously

Sponsors & Collaborators

  • Homology Medicines, Inc

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
45 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-09-08
Primary Completion
2024-10-31
Completion
2029-01-31
FDA Drug
Yes

Countries

  • United States
  • Canada

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05238324 on ClinicalTrials.gov