Observational study of patients treated with a matched unrelated donor
Research type
Research Study
Full title
A Prospective observational study of paediatric patients affected by haematological disorders treated with matched unrelated donor stem cell transplant
IRAS ID
230731
Contact name
Waseem Qasim
Contact email
Sponsor organisation
Bellicum Pharmaceuticals, Inc.
Duration of Study in the UK
0 years, 9 months, 1 days
Research summary
This is a prospective observational study of Paediatric patients affected by haematological disorders who do not have a matched related donor (optimal standard of care) but are treated with matched unrelated donor (MUD) stem cell transplantation (9/10 or 10/10 HLA match).
Patients will be enrolled regardless of disease, malignant diseases include high-risk ALL in 1st CR, ALL in 2nd or subsequent CR, high-risk AML in 1st CR, AML in 2nd CR, myelodysplastic syndromes. Also non-malignant diseases which include primary immune deficiencies, osteopetrosis, inherited bone marrow failure disorders selected cases of erythroid disorders such as β0 β0 thalassemia major, sickle cell disease, Diamond-Blackfan anaemia congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant evolution (MDS, AML).
After informed consent, the patients will be screened against the inclusion/exclusion criteria, and if meeting the inclusion/exclusion criteria, the subject will be enrolled and data collected.
Up to 30 patients from the ages of 0-18 years will be enrolled onto the study. The minimum study data collection for each patient will be for 180 days post-transplant. The data collected for these patients will be used to serve as a comparison to the BP-004 protocol in which paediatric patients with haematologic disorders who do not have a matched related, or matched unrelated donor undergo haploidentical transplantation HSCT followed by BPX-501 T cell addback.REC name
London - South East Research Ethics Committee
REC reference
17/LO/1571
Date of REC Opinion
18 Sep 2017
REC opinion
Favourable Opinion