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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

Study Summary

This trial will test if a single dose of HMI-103 is safe and effective in adults with classical PKU due to PAH deficiency.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
To evaluate the efficacy of HMI-103 on reduction of plasma Phe concentration at each dose level
To measure incidence and severity of Treatment Emergent Adverse Events (TEAEs) and adverse events of special interest (AESIs) of a single administration of HMI-103
Secondary outcome measures
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

Frequently Asked Questions

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

Does this research trial permit seniors to participate?

"This trial is restricted to individuals aged 18-55. However, there are 14 clinical studies for minors and 12 for those beyond the age of 65."

Answered by AI

How detrimental is HMI-103 to patient health?

"The safety of HMI-103 is estimated to be a 1 due to its Phase 1 trial status, which suggests there is limited data regarding both efficacy and safety."

Answered by AI

What is the current participant allotment for this investigation?

"To fully partake in this trial, 9 qualified participants meeting the inclusion criteria are needed. Patients can join from CHOC Children's Health of Orange County located in California or UPMC Children's Hospital of Pittsburgh situated in Michigan."

Answered by AI

What is the scope of implementation for this clinical trial across healthcare facilities?

"The 5 trial sites for this medical study are situated in Orange, Pittsburgh and Grand Rapids plus two other cities. For the sake of convenience, it is encouraged to pick a clinic close to you if you choose to participate."

Answered by AI

Is there still capacity for individuals to join this investigation?

"Clinicaltrials.gov states that this medical research is currently in the process of sourcing participants; its first appearance was on June 3rd 2022, and it was revised as recently as October 26th 2022."

Answered by AI

Am I qualified to join this experimental program?

"In total, 9 participants who suffer from phenylketonurias and range in age between 18 to 55 are being recruited for this trial. In order to qualify, applicants must have a minimum plasma Phe concentration of 600 μmol/L supported by at least four measurements within the past 24 months. Furthermore, they need to demonstrate an inability to regulate their PKU disease through dietary management as judged by both the investigator and DMC. As part of the study conditions, patients will be required to adhere strictly to their baseline diet unless explicitly instructed otherwise."

Answered by AI

Who else is applying?

What state do they live in?
Michigan
How old are they?
< 18
What site did they apply to?
Spectrum Health Butterworth Hospital
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency
2022. "Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05222178.
~1 spots leftby Apr 2025
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