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Clinical Trial Summary

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.


Clinical Trial Description

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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT02354443
Study type Interventional
Source Fate Therapeutics
Contact
Status Terminated
Phase Phase 1
Start date June 2015
Completion date February 2017

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