NCT02354443

A Trial of a Single ProHema-CB Product Transplant in Pediatric Patients With Inherited Metabolic Disorders

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

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Fate Therapeutics · Industry
• Sex: All
• Age: 1 Year – 18 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Metabolic Disorders

Interventions

Type	Name	Description
BIOLOGICAL	ProHema-CB Transplant	ProHema-CB, the cellular product, represents the cell populations contained within a human UCB unit after modulation on the day of transplantation by an ex vivo incubation process with the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 (also referred to as FT1050). The cell populations include hematopoietic stem and progenitor cells.

Timeline

• Start date: 2015-06-01
• Primary completion: 2016-12-01
• Completion: 2017-02-01
• First posted: 2015-02-03
• Last updated: 2018-10-10
• Results posted: 2018-10-10

Locations

2 sites across 1 country: United States