Metabolic Disorders Clinical Trial
Official title:
A Phase 1 Trial of a Single ProHema® CB Product as Part of Single Cord Blood Unit Transplant After Busulfan/Cyclophosphamide/ATG Conditioning for Pediatric Patients With Inherited Metabolic Disorders
The purpose of this study is to describe the safety profile of ProHema-CB as part of a single cord blood unit transplant after a myeloablative conditioning regimen in pediatric patients with inherited metabolic disorders. The safety profile will primarily be assessed by neutrophil engraftment.
This study is an open-label trial of the safety of a single cord blood transplant using
ProHema-CB following busulfan/cyclophosphamide/ATG conditioning for pediatric patients with
inherited metabolic disorders.
A maximum of 12 eligible male and female subjects (1 to 18 years old, inclusive) will be
enrolled and treated in the trial at approximately 1 to 3 centers within the U.S.
All subjects will be admitted to the hospital, per institutional practice and will receive a
conditioning regimen, after which they will receive a HLA-matched or partially matched
ProHema -CB unit on Day 0.
They will receive study follow up assessments weekly following Day 0 through Day 100 and
study visit Days 180, 270, 365 and 730.
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