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Gene Editing
Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency
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
No Placebo-Only Group
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)
- Tetrahydrobiopterin Deficiency
- 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
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
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
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
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.