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

N/AEnrolling By InvitationNCT04348708

Homology Medicines, Inc21 enrolled

Overview

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

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

Study Type

OBSERVATIONAL

Enrollment

Conditions

Phenylketonuria PAH Deficiency

Interventions

HMI-102GENETIC

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

Eligibility

Sex: ALLMin age: 18 YearsMax age: 55 Years

Medical Language ↔ Plain English

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.

Locations (2)

Boston Children's Hospital

Boston, Massachusetts, United States

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Outcomes

Primary Outcomes

Incidence and severity of adverse events of special interest (AESIs) and serious adverse events (SAEs) related to HMI 102

Subjects with at least one AESI or SAE