Status:
TERMINATED
Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency
Lead Sponsor:
Homology Medicines, Inc
Conditions:
Phenylketonurias
PAH Deficiency
Eligibility:
All Genders
18-55 years
Phase:
PHASE1
Brief Summary
This is an open-label, sequential ascending dose-escalation, Phase 1 study to evaluate the safety and efficacy of a single intravenous (I.V.) administration of HMI-103, a gene editing development cand...
Detailed Description
This is an open-label, sequential ascending dose-escalation, Phase 1 study to evaluate the safety and efficacy of a single intravenous administration of HMI-103, a gene editing development candidate, ...
Eligibility Criteria
Inclusion
- Adults 18-55 years of age at the time of informed consent
- Diagnosis of classical phenylketonuria (PKU) due to PAH deficiency
- Four baseline plasma Phe values with a concentration of ≥ 600 μmol/L and at least one historical value ≥ 600 μmol/L in the preceding 24 months.
- Participants must have uncontrolled classical PKU disease (despite Phe-restricted dietary management) in the judgment of the investigator and confirmed by the independent DMC at the end of the Screening period.
- Participant has the ability and willingness to maintain their baseline diet, for the duration of the trial, unless otherwise directed
Exclusion
- Subjects with PKU that is not due to PAH deficiency
- Presence of anti-AAVHSC15 neutralizing antibodies
- Participants who are well controlled on a Phe-restricted diet.
- Hemoglobin A1c \>6.5% or fasting glucose \>126 mg/dL
- Liver function tests \> ULN
- International normalized ratio (INR) \> 1.2
- Hematology values outside of the normal range
- Previously received gene therapy for the treatment of any condition.
Key Trial Info
Start Date :
June 3 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 14 2023
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT05222178
Start Date
June 3 2022
End Date
September 14 2023
Last Update
October 10 2023
Active Locations (2)
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1
The Community Health Clinic
Topeka, Indiana, United States, 46571
2
Clinic for Special Children
Lancaster, Pennsylvania, United States, 17579