Clinical Trial: Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Study Evaluating BPX-501 T Cells and AP1903 for Prevention of Graft Versus Host Disease (GVHD) After Haploidentical, Related, T Cell-Depleted Hematopoietic Cell Transplantation for Non-Malignant Dis

Brief Summary: The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of AP1903 on day 7 to prevent GVHD.

Detailed Summary: This is a single arm dose finding study evaluating the safety and efficacy of a BPX 501 infusion (T cells genetically modified with the inducible Caspase 9 suicide gene) of 3x10E6 to 1X10E7 cells/kg followed by an AP1903 infusion on day 7 after a partially mismatched, related, T cell-depleted hematopoietic cell transplantation (HCT) in patients with non-malignant diseases. The purpose of this clinical trial is to determine the dose of BPX 501 T cell infusion with subsequent planned infusion of AP1903 which can facilitate engraftment and prevent the occurrence of GVHD.
Sponsor: Bellicum Pharmaceuticals

Current Primary Outcome: GVHD [ Time Frame: day 100 ]

The incidence and Grade of acute GVHD will be determined


Original Primary Outcome: Same as current

Current Secondary Outcome: Transplant related mortality [ Time Frame: 6 months ]

Original Secondary Outcome: Same as current

Information By: Bellicum Pharmaceuticals

Dates:
Date Received: August 28, 2014
Date Started: February 2015
Date Completion: December 2019
Last Updated: March 31, 2017
Last Verified: October 2016