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Gene Editing

HMI-103 for Pyridoxine-Dependent Epilepsy

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 weeks 48-52
Awards & highlights

Summary

This trial tests HMI-103, a gene editing treatment, in adults with uncontrolled PKU due to PAH deficiency. The treatment aims to fix the faulty gene so the body can process phenylalanine properly.

Eligible Conditions
  • Pyridoxine-Dependent Epilepsy (PDE)
  • Phenylketonuria

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~weeks 48-52
This trial's timeline: 3 weeks for screening, Varies for treatment, and weeks 48-52 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
To evaluate the efficacy of HMI-103 on reduction of plasma Phe concentration at each dose level
Secondary study objectives
To assess durability of response
To assess the changes in dietary protein intake
To evaluate the effect of HMI-103 on plasma Phe concentration relative to treatment guidelines for PKU

Trial Design

3Treatment groups
Experimental Treatment
Group I: Low Dose CohortExperimental Treatment1 Intervention
HMI-103 delivered IV one time
Group II: Intermediate Dose CohortExperimental Treatment1 Intervention
HMI-103 delivered IV one time
Group III: High Dose CohortExperimental Treatment1 Intervention
HMI-103 delivered IV one time

Find a Location

Who is running the clinical trial?

Homology Medicines, IncLead Sponsor
5 Previous Clinical Trials
12,038 Total Patients Enrolled

Media Library

HMI-103 (Gene Editing) Clinical Trial Eligibility Overview. Trial Name: NCT05222178 — Phase 1
Pyridoxine-Dependent Epilepsy Research Study Groups: High Dose Cohort, Low Dose Cohort, Intermediate Dose Cohort
Pyridoxine-Dependent Epilepsy Clinical Trial 2023: HMI-103 Highlights & Side Effects. Trial Name: NCT05222178 — Phase 1
HMI-103 (Gene Editing) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05222178 — Phase 1
~1 spots leftby Oct 2025