A Phase 2/3 interventional study of Fludarabine and Thiotepa in Chronic Myeloid Leukemia, sponsored by St. Anna Kinderkrebsforschung. Completed at 4 sites in 3 countries. Open to participants aged 1 Year to 18 Years. Per ClinicalTrials.gov, last updated 2021-04-15.
Sponsored by St. Anna Kinderkrebsforschung · Phase 2/3, Interventional, and Treatment
In children and adolescents with chronic myeloid leukaemia (CML) stem cell transplantation (SCT) may be a valid alternative to the life-long treatment with tyrosinkinase inhibitors (TKI). This trial aims to evaluate the use of a reduced intensity conditioning regimen (RIC), consisting of fludarabine, melphalan and thiotepa in order to minimize transplant related mortality and toxic late effects. Strict post-transplant monitoring and reintroduction of TKI as well as donor lymphocyte infusions (DLI) in case of relevant residual disease are part of the protocol.
Chronic myeloid leukaemia (CML) is a rare disease in children with an incidence of 3-5% of all paediatric leukaemias. Since the introduction of tyrosinkinase inhibitors (TKI) stem cell transplantation (SCT) is no longer the first choice treatment for patients with early phase CML.
However life-long treatment with TKI may not be feasable in several cases due to side effects such as growth retardation, non-compliance and resistance. This protocol evaluates the feasibility of SCT following a reduced intensity conditioning regimen (RIC) consisting of fludarabine, melphalan, thiotepa and thymoglobuline (ATG). Matched siblings and matched unrelated donors are permitted for stem cell donation. In case of unrelated donors tissue typing has to be done by high resolution molecular typing. Donors with 10/10 or 9/10 identical allels in the human leukocyte antigen (HLA) system are accepted. Preferred stem cell source is bone marrow but peripheral blood stem cells and umbilical cord blood are also allowed. Graft-versus-Host-Disease (GvHD)-prophylaxis is achieved with cyclosporine A and mycophenolate mofetil.
Monitoring of the breakpoint cluster region - Abelson (BCR/ABL) rearrangement is performed monthly in the first year after SCT. In case of BCR/ABL positivity TKI are given in the first year after SCT. Followed by donor lymphocyte infusions (DLI) later on if BCR/ABL positivity persists.
894 studies on the registry are indexed under Leukemia, Myelogenous, Chronic, BCR-ABL Positive; 109 are open to participants now.
This study's enrollment of 13 is below the median of 44 across 681 interventional studies indexed under Leukemia, Myelogenous, Chronic, BCR-ABL Positive.
Browse Leukemia, Myelogenous, Chronic, BCR-ABL Positive studies →St. Anna Kinderkrebsforschung is the lead sponsor of 12 studies on the registry; 2 are open to participants now.
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Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
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Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Fludarabine intravenous - daily dose: 40mg/sqm on day -7, -6, -5, -4; Thiotepa intravenous - daily dose: 2 x 5mg/kg on day -3; Melphalan intravenous - daily dose: 140/mg/sqm on day - 2; ATG intravenous - dose according to local standards on day -3, -2, -1; bone marrow or peripheral blood stem cells of an HLA identical sibling or matched unrelated donor on day 0; GvHD propyhlaxis with Mycophenolate Mofetil and Cyclosporine A
Drug: Fludarabine · Drug: Thiotepa · Drug: Melphalan · Drug: ATG · Drug: Cyclosporine A · Drug: Mycophenolate mofetil · Biological: bone marrow or peripheral blood stem cells
infusion
Also known as: Fludara
infusion
Also known as: Thioplex, TESPA
infusion
Also known as: Alkeran
infusion
Also known as: Thymoglobulin
infusion, orally if possible
Also known as: Sandimmune
infusion
Also known as: CellCept
infusion
transplant related mortality
Time frame: one year
overall survival
Time frame: five years
event free survival
Time frame: five years
Plan to share: Undecided
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This study is completed, as verified in Apr 2021. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Fludarabine · Thiotepa · Melphalan · Antilymphocyte Serum · Cyclosporine · Mycophenolic Acid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive →
St. Anna Kinderkrebsforschung