Phase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders


3 United Kingdom sites

1 Italy site


1 to 18 Years


Phase 2

Study type









About the study

This study will evaluate pediatric patients with malignant or non-malignant blood cell
disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa
and beta cells that comes from a partially matched family donor. The study will assess
whether immune cells, called T cells, from the family donor, that are specially grown in the
laboratory 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
programmed with a self-destruct switch so that they can be destroyed if they start to react
against tissues (Graft versus host disease).

participation requirements

– Age < 18 years and > 1 month (< 1 month upon approval by Sponsor) - Life expectancy > 10 weeks

– Patients deemed clinically eligible for allogeneic stem cell transplantation.

– Patients may have failed prior allograft

– Patients with life-threatening acute leukemia (high-risk ALL in 1st CR, ALL in 2nd CR,
high-risk AML in 1st CR, AML in 2nd CR.) or myelodysplastic syndromes. Morphological
CR must be documented and minimal residual disease measurement before transplantation
is recommended.

– Non-malignant disorders deemed curable by allogeneic transplantation: a. primary
immune deficiencies, b. severe aplastic anemia not responding to immune suppressive
therapy, c. osteopetrosis d. selected cases of erythroid disorders such as β0 β0
thalassemia major, sickle cell disease, Diamond-Blackfan anemia.

e. congenital/hereditary cytopenia, including Fanconi Anemia before any clonal
malignant evolution (MDS, AML).

Note: Subjects will be eligible if they meet either item 5 OR item 6.

– Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically
identical relative or 10/10 unrelated donor evaluated using high resolution molecular
typing) or presence of rapidly progressive disease not permitting time to identify an
unrelated donor

– A minimum genotypic identical match of 5/10 is required.

– The donor and recipient must be identical, as determined by high resolution typing, on
at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA-
DRB1 and HLA-DQB1.

– Lansky/Karnofsky score > 50

– Signed informed consent by the patient or the patient’s parent or guardian for
patients who are minors

participation restrictions

– Greater than active Grade II acute GvHD or chronic extensive GvHD due to a previous
allograft at the time of screening

– Patient receiving an immunosuppressive treatment for GvHD treatment due to a previous
allograft at the time of screening

– Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal
value), or of renal function (creatinine clearance <30ml/min/1.73m2) - Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 40%) - Current clinically active infectious disease (including positive HIV serology or viral RNA) - Serious concurrent uncontrolled medical disorder - Pregnant or breast feeding female patient - Lack of parents'/guardian's informed consent for children who are minors.


  • Roma, Italy, IRCCS Ospedale Pediatrico Bambino Gesù, 00161
  • London, United Kingdom, Royal Free London NHS Foundation Trust, NW3 2QG
  • London, United Kingdom, Institute of Child Health & Great Ormond Street Hospital, WC1N 1EH
  • Newcastle Upon Tyne, United Kingdom, Great North Children's Hospital, NE1 4LP