CaspaCide TCR αβ haplo HSCT
Research type
Research Study
Full title
Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR αβ+ T cells in pediatric patients affected by hematological disorders
IRAS ID
170965
Contact name
Annemarie Moseley
Contact email
Sponsor organisation
Bellicum Pharmaceuticals, Inc.
Eudract number
2014-000584-41
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
15 years, 0 months, 1 days
Research summary
This study will evaluate patients (aged from 3 months up to 21 years) with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant. The study will assess whether immune cells, called T cells, from a partially matched family donor, that are specially grown in the laboratory and genetically modified (BPX-501) and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been modified with a self-destruct switch so that they can be destroyed if they start to react against the tissues, a condition called Graft versus Host Disease (GVHD).
The study will test whether BPX-501 infusion can improve the patient’s immune recovery. The study will also look at the potential of reducing the severity and duration of GVHD. If the patient experiences GVHD following treatment with BPX-501, they may be treated with an infusion of AP1903. AP1903 is a drug that has been designed to activate the self-destruct switch in BPX-501 to stop the effects of the treatment. AP1903 will only be given to patients with GVHD who do not respond within 7 days to standard of care treatment.
The study will initially look at 3 different increasing doses of the study product (BPX-501). When the highest safe dose for BPX-501 is found, further patients will be treated with this dose. Safety and survival over 180 days and 1 year will be assessed for all patients. The study will look at the safety of both BPX-501 and AP1903 as well as the efficacy of BPX-501.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
15/LO/0618
Date of REC Opinion
29 May 2015
REC opinion
Further Information Favourable Opinion