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

Homology Medicines

Status: Invitation-only

Conditions: Phenylketonuria; PAH Deficiency

Treatments: Genetic: HMI-102

Study type: Observational

Funder types: Industry

Identifiers

NCT04348708

HMI-102-102

Details and patient eligibility

About

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

Full 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

Enrollment: 21 estimated patients

Sex: All

Ages: 18 to 55 years old

Volunteers: No Healthy Volunteers

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.

Trial design

21 participants in 3 patient groups

Dose Level Cohort 1

Description:

Dose Level 1 of HMI-102 delivered intravenously one time

Treatment:

Genetic: HMI-102

Dose Level Cohort 2

Description:

Dose Level 2 of HMI-102 delivered intravenously one time

Treatment:

Genetic: HMI-102

Dose Level Cohort 3

Description:

Dose Level 3 of HMI-102 delivered intravenously one time

Treatment:

Genetic: HMI-102