Use of parental stem cells for children requiring a bone marrow transplant
TCR a+ß+/CD19+ cell depleted haploidentical donor stem cell transplantation for paediatric patients
Kids Oncology And Leukaemia TriAls (KOALA)
94 participants
Sep 7, 2018
Interventional
Conditions
Summary
The primary purpose of this trial is to evaluate the efficacy of using parent donors for bone marrow transplants in children, using a particular processing method of the donor blood cells (TCR a+ß+/CD19+ cell depletion) compared to those receiving a standard bone marrow transplant from another donor, We also want to compare cost to the health system of TCR a+ß+/CD19+ cell depletion with that of standard bone marrow transplant procedures, Who is it for? Patients may be eligible to enrol in this trial if they are aged 3 months to 18 years of age, have been diagnosed with haematological malignancies or non malignant disorders requiring a bone marrow transplant, and have an eligible parent donor. Study details Patients who have no fully matched brothers or sisters, and no well matched volunteer donors or umbilical cord blood units available will receive a TCR a+ß+/CD19+ cell depleted graft from a parent donor. Control patients will receive a standard bone marrow transplant from another donor (e.g., volunteer donor) as per institutional practice. Patients will undergo clinical assessments at regular intervals for two years following the transplant to evaluate the efficacy of the treatment. Further information, such as quality of life and carer burden, will be collected for up to 5 years following the transplant. It is hoped that the findings from this trial will provide information on whether parent donor cells undergoing specialised cell processing can be effectively used for bone marrow transplants in children with haematological malignancies or non malignant disorders.
Eligibility
Inclusion Criteria11
- A patient must meet the following criteria to be eligible for inclusion of Arm 1 of the study:
- Aged less than 18 years
- The availability of a HLA matched haploidentical donor greater or equal to 5/10
- Absence of readily available matched sibling donor
- Absence of other readily available non-related donors:
- Unrelated volunteer donors; HLA match less than 9/10
- UCB units with adequate cell dose; HLA match less than 5/6 on low resolution typing or HLA match less than 6/8 on high resolution typing (8/8 HLA match for non malignant patients)
- Adequate organ function
- Female patients of child bearing age are not pregnant
- Written consent of the patient’s legal guardian, and assent from the patient if age appropriate
- All potential parent donors must undergo high resolution HLA typing, and assessment as to their fitness to undergo peripheral blood stem cell collection via G-CSF stimulated apheresis.
Exclusion Criteria4
- Patient has undergone HSCT within the last 180 days
- Evidence of HIV infection
- Current uncontrolled bacterial or fungal infection
- The treatment protocol in part or in its entirety is declined by either the patient or their legal guardian
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Interventions
Arm 1 - Group receiving investigational device Patients will receive a TCR a+ß+/CD19+ cell depleted graft from a haploidentical parental donor. Candidates will only be eligible for Arm 1 of the study if there are no suitable human leucocyte antigen (HLA) matched readily available donors, and no suitably matched umbilical cord blood units available. Conditioning therapy used will be dependent on HSCT indication. GVHD prophylaxis will include in vivo serotherapy (ATG) (once daily IV dose of 5mg/kg for 3 days) and mycophenolate mofetil in non-malignant patients (15mg/kg 3 times a day -2 to day +30, given initially IV with an option to switch to orally dependent on patient tolerability). Donor product will be peripheral blood stem cells collected via G-CSF stimulated apheresis. Apheresis will be performed as per FACT guidelines, national standards and individual institution protocols. All donor products will be processed in FACT accredited Bone Marrow Transplant laboratories. Fresh donor product will undergo TCR aß+ depletion and CD19+ depletion using Miltenyi CliniMACS® paramagnetic bead technology. The target CD34+ dose will be 10 x10^6/kg recipient weight, with a recommended minimal dose of 5 x 10^6/kg. Patients on Arm 1 will be assessed for neutrophil and platelet engraftment, transplant related mortality, graft versus host disease, relapse and overall survival. They will also be assessed for immune reconstitution, economic and quality of life outcomes. Arm 2 – Control group Candidates for Arm 2 will be patients undergoing alternate donor HSCT, defined as any donor that is not an HLA matched sibling (e.g., volunteer donor, matched umbilical cord blood units). Economic and Quality of Life outcomes will be measured for these patients post-transplant.
Locations(6)
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ACTRN12617001174370