NCT05222178

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

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

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Homology Medicines, Inc · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

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.

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, 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.

Conditions

• Phenylketonurias
• PAH Deficiency
• Phenylketonuria

Interventions

Type	Name	Description
DRUG	HMI-103	HMI-103 is an AAVHSC15 capsid containing a functional copy of the human PAH gene

Timeline

• Start date: 2022-06-03
• Primary completion: 2023-09-14
• Completion: 2023-09-14
• First posted: 2022-02-03
• Last updated: 2023-10-10

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study