Safety and Efficacy of HMI-103, a Gene Editing Development Candidate in Adults With Classical PKU Due to PAH Deficiency
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- STATUS
- Recruiting
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- End date
- Jun 11, 2028
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- participants needed
- 9
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- sponsor
- Homology Medicines, Inc
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 Identifier | NCT05222178 |
| Sponsor | Homology Medicines, Inc |
| Last Modified on | 4 October 2022 |
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