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Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

Phenylketonuria Clinical Trial

Official title:
A Phase 1, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of HMI-103 Administered Intravenously in Adult Participants With Classical PKU Due to PAH Deficiency

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05222178
Study type Interventional
Source Homology Medicines, Inc
Contact
Status Terminated
Phase Phase 1
Start date June 3, 2022
Completion date September 14, 2023
View Details
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