10
Participants
Start Date
June 10, 2019
Primary Completion Date
January 10, 2023
Study Completion Date
August 1, 2023
HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene
HMI-102
"Control subjects will generally have the same assessments as treated subjects.~Control subjects will undergo pre-baseline procedures to confirm that they are eligible to receive treatment with HMI-102. Once eligible control subjects are dosed with HMI-102, they will initiate the same post-dose procedures as subjects who received HMI-102."
Icahn School of Medicine at Mount Sinai, New York
UPMC Children's Hospital of Pittsburgh, Pittsburgh
Children's Hospital of Philadelphia, Philadelphia
The University of North Carolina At Chapel Hill, Chapel Hill
Emory University Hospital, Atlanta
University of South Florida, Tampa
Nationwide Children's Hospital, Columbus
Lurie Children's Hospital of Chicago, Chicago
University of Texas Southwestern Medical Center, Dallas
University of Utah, Salt Lake City
Kaiser Permanente Los Angeles Medical Center, Los Angeles
Children's Hospital of Orange County, Orange
Boston Children's Hospital, Boston
Lead Sponsor
Homology Medicines, Inc
INDUSTRY