Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency
Stopped early · Phase 1
Conditions studied: Phenylketonurias, PAH Deficiency, Phenylketonuria
In brief
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.
Key facts
- Study ID
- NCT05222178
- Run by
- Homology Medicines, Inc
- People needed
- 3
- Starts
- 2022-06-03
- Expected to finish
- 2023-09-14
- Last updated by the study team
- 2023-10-10
Who can join
Age: 18 and older, up to 55. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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.
Where it is running
- The Community Health Clinic — Topeka, Indiana, United States
- Clinic for Special Children — Lancaster, Pennsylvania, United States
Full record on ClinicalTrials.gov
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