NCT04348708

Long-Term Follow Up Study of Subjects Previously Administered HMI 102

An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects With PAH Deficiency Previously Administered HMI 102

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 21 (estimated)
• Sponsor: Homology Medicines, Inc · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects with PAH Deficiency Previously Administered HMI 102

Detailed description

This Long-Term Follow Up Study will evaluate the safety and efficacy of PKU Subjects with PAH Deficiency Previously Administered HMI 102. Subjects will have already received a single dose of HMI-102 administered intravenously

Conditions

• Phenylketonuria
• PAH Deficiency

Interventions

Type	Name	Description
GENETIC	HMI-102	HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene

Timeline

• Start date: 2020-08-19
• Primary completion: 2026-01-30
• Completion: 2026-12-01
• First posted: 2020-04-16
• Last updated: 2022-06-30

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study