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ENROLLING BY INVITATION
NCT04348708
Long-Term Follow Up Study of Subjects Previously Administered HMI 102
Sponsor: Homology Medicines, Inc
View on ClinicalTrials.gov
Summary
An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects with PAH Deficiency Previously Administered HMI 102
Official title: An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects With PAH Deficiency Previously Administered HMI 102
Key Details
Gender
All
Age Range
18 Years - 55 Years
Study Type
OBSERVATIONAL
Enrollment
21
Start Date
2020-08-19
Completion Date
2026-12-01
Last Updated
2022-06-30
Healthy Volunteers
No
Conditions
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, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States