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

Homology Medicines, Inc

Summary

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

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

Eligibility

Age range: 18–55 years
Sex: All
Healthy volunteers: No

Detailed criteria

Inclusion Criteria: * Subject was previously administered HMI 102. * Subject is able to understand the purpose and risks of the study and is willing to provide informed consent. * Subject is able to comply with all study procedures and long-term follow-up. Exclusion Criteria: * Participation in the study is not in the subject's best interest, in the opinion of the Investigator.

Interventions

Genetic
HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene

Locations (2)

Boston Children's Hospital
Boston, Massachusetts
Icahn School of Medicine at Mount Sinai
New York, New York