Description
Seeking people aged 18-75 with Facioscapulohumeral muscular dystrophy (FSHD) for a study of an investigational gene and cell therapy infusion.
Overview
Participants in this study will get a one-time infusion of the investigational gene and cell therapy during a several-day hospital stay. Following hospital discharge, participants will need to be within a 1-hour drive of the hospital for 2 weeks. There will be about 12 study visits in the first year. For a few months following the infusion participants will need to take prednisone.
What we're hoping for
We are studying if an investigational gene and cell therapy infusion is safe and if it helps adults with Facioscapulohumeral muscular dystrophy (FSHD) function better.
Additional Information
ClinicalTrials.gov Identifier: NCT06907875