Safety and Efficacy of HMI-103, a Gene Editing Development Candidate in Adults With Classical PKU Due to PAH Deficiency

  • STATUS
    Recruiting
  • End date
    Jun 11, 2028
  • participants needed
    9
  • sponsor
    Homology Medicines, Inc
Updated on 4 October 2022
deficiency
pah gene

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 candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe restricted dietary management.

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, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe-restricted dietary management. Up to 3 dose levels of HMI-103 may be investigated. At a given dose level, 3 participants are planned to be enrolled and dosed. Participant dosing will be staggered. Following evaluation of data from the first 3 participants in a cohort, the DMC will decide to escalate to the next dose level or expand the cohort at the selected dose level.

Details
Condition Phenylketonurias, PAH Deficiency, Phenylketonuria
Treatment HMI-103
Clinical Study IdentifierNCT05222178
SponsorHomology Medicines, Inc
Last Modified on4 October 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

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 Criteria

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
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