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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 weeks3 visits
Treatment ~ Varies
Follow Up ~ weeks 48-52
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~weeks 48-52
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
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Who is running the clinical trial?
Homology Medicines, IncLead Sponsor
5 Previous Clinical Trials
12,038 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- You have received gene therapy in the past for any medical condition.You have a minimum age of 18 years.\nYou have a PAH deficiency due to a PAH deficiency.You have the ability and willingness to maintain your baseline diet, for the duration of the trial, unless otherwise directed.
Research Study Groups:
This trial has the following groups:- Group 1: High Dose Cohort
- Group 2: Low Dose Cohort
- Group 3: Intermediate Dose Cohort
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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