Clinical trial NCT01423500: ALL-SCT BFM International- HSCT in Children and Adolescents With ALL
Official title: Allogeneic Stem Cell Transplantation in Children and Adolescents With Acute Lymphoblastic Leukaemia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Lymphoblastic Leukemia, Acute, Childhood;
Keywords: ALL
HSCT
children
adolescents
Lead sponsor: St. Anna Kinderkrebsforschung
Sponsor class: OTHER
First posted: 2011-08-26
Enrollment: 405
Sex: ALL
Minimum age (years): 3
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: With this protocol the ALL-SCT BFM international study group wants

* to evaluate whether hematopoietic stem cell transplantation (HSCT) from matched family or unrelated donors (MD) is equivalent to the HSCT from matched sibling donors (MSD).
* to evaluate the efficacy of hematopoietic stem cell transplantation (HSCT)from mismatched family or unrelated donors (MMD) as compared to HSCT from matched sibling donors or matched donors.
* to determine whether therapy has been carried out according to the main HSCT protocol recommendations. The standardisation of the treatment options during HSCT from different donor types aims at the achievement of an optimal comparison of survival after HSCT with survival after chemotherapy only.
* to prospectively evaluate and compare the incidence of acute and chronic Graft-versus-Host-Disease (GvHD) after HSCT from matched sibling donor (MSD), from matched donor (MD) and from mismatched donor (MMD).

Detailed description: Patients with high risk or relapsed acute lymphoblastic leukaemia (ALL) have a worse prognosis compared to all other patients with ALL. For these patients additional therapy approaches are required after they have achieved remission with multimodal chemotherapy. Allogeneic haematopoetic stem cell transplantation shows promising results mainly due to an immunological antileukaemic control by the graft-versus-leukaemia effect but treatment related mortality and morbidity remains a serious problem.

Eligibility criteria: Inclusion Criteria:

* age at time of initial diagnosis or relapse diagnosis, respectively under or equal 18 years
* indication for allogeneic hematopoietic stem cell transplantation(HSCT)
* complete remission before hematopoietic stem cell transplantation (HSCT)
* written consent of the parents (legal guardian) and, if necessary, the minor patient via Informed Consent Form
* no pregnancy
* no secondary malignancy
* no previous hematopoietic stem cell transplantation (HSCT)
* hematopoietic stem cell transplantation (HSCT) is performed in a study participating centre.

Exclusion Criteria:

* age at time of initial diagnosis or relapse diagnosis, respectively above 18 years
* no indication for allogeneic HSCT
* no complete remission before SCT
* no written consent of the parents (legal guardian) and, if necessary, the minor patient via Informed Consent Form
* pregnancy
* secondary malignancy
* previous HSCT
* HSCT is not performed in a study participating centre.

Primary outcomes:
measure: Event free survival
timeFrame: 10 years
Secondary outcomes:
measure: number of patients with GvHD acute and chronic Graft-versus-Host-Disease (GvHD)
timeFrame: 10 years
measure: occurrence and course of late effects after chemotherapy with subsequent allogeneic HSCT
timeFrame: 10 years
measure: occurrence and course of late effects after chemotherapy with subsequent allogeneic HSCT
timeFrame: 10 years
measure: occurrence and course of late effects after chemotherapy with subsequent allogeneic HSCT
timeFrame: 10 years
measure: occurrence and course of subsequent malignancies after chemotherapy with subsequent allogeneic HSCT
timeFrame: 10 years
Other outcomes:
Locations:
city: Graz
country: Austria
facility: Universitätsklinik für Kinder- und Jugendheilkunde, Klin. Abt. f. Hämato-Onkologie
city: Innsbruck
country: Austria
facility: Universitätsklinik für Kinder- und Jugendheilkunde
city: Vienna
country: Austria
facility: St. Anna Children's Hospital
city: Prague
country: Czech Republic
facility: Department of Paediatric Haematology and Oncology HSCT-Unit
city: Copenhagen
country: Denmark
facility: Pediatric Clinic II, Rigshospitalet
city: Paris
country: France
facility: Pediatric Immuno-Hematology Unit Robert Debré Hospital
city: Haifa
country: Israel
facility: Rambam Medical Center
city: Petach-Tikva
country: Israel
facility: Schneider Children's Medical Center of Israel
city: Monza
country: Italy
facility: Clinica Pediatrica dell'Universita di Milano Bicocca, Hospitale San Gerardo
city: Leiden
country: Netherlands
facility: Leiden University Hospital
city: Nijmegen
country: Netherlands
facility: Radboud University - Nijmegen Medical Centre
city: Utrecht
country: Netherlands
facility: Department of Paediatric Haematology and Oncology, Wilhelmina Children's Hospital
city: Bydgoszcz
country: Poland
facility: University Hospital, Collegium Medicum UMK, Pediatric Hematology and Oncology
city: Cracow
country: Poland
facility: Department of Transplantation, University Children's Hospital
city: Lublin
country: Poland
facility: Children's University Hospital - Hematology - Oncology
city: Poznan
country: Poland
facility: Department of Pediadric Oncology, Hematology and Transplantology, University of Medical Sciences
city: Wroclaw
country: Poland
facility: Wroclaw Medical University, Dept. of Children Hematology and Oncology
city: Bratislava
country: Slovakia
facility: Department of Pediatric Bone Marrow Transplantation Unit, University Childrens´ Hospital
city: Lund
country: Sweden
facility: Department of Pediatric Oncology, Lund University Hospital
city: Ankara
country: Turkey
facility: Department of Pediatrics, Gülhane Military Medical Academy
city: Ankara
country: Turkey
facility: Dept. of Paediatrics - BMT Unit, School of Medicine, University of Ankara
city: Antalya
country: Turkey
facility: Department of Pediatric Hematology-Oncology and Pediatric Stem Cell Transplantation, Akdeniz University School of Medicine
city: Istanbul
country: Turkey
facility: Department of Pediatric Hematology, Oncology and BMT, Istanbul School of Medicine
city: Izmir
country: Turkey
facility: Pediatric BMT Centre, Ege University
Study officials:
affiliation: St. Anna Kinderkrebsforschung
name: Christina Peters Peters, Prof MD PHD
role: STUDY_CHAIR
affiliation: Department of Paediatric Haematology and Oncology. HSCT Unit Prague
name: Petr Sedlacek, Prof. MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Paediatric Clinic II, Rigshospitalet Copenhagen
name: Marianne Ifversen, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: HSCT Unit Robert Debré Hospital Paris
name: Jean-Hugues Dalle, Prof. MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Schneider Children's Medical Center, Israel
name: Jerry Stein, Prof. MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Ospedale San Gerardo Monza
name: Adriana Balduzzi, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Wilhelmina Children's Hospital Utrecht
name: Marc Bierings, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Department of Paediatric Oncology, Haematology and Transplantology, University of Medical Sciences Poznan
name: Jacek Wachowiak, MD, Prof.
role: PRINCIPAL_INVESTIGATOR
affiliation: HSCT Unit, University Children's Hospital Bratislava
name: Sabina Sufliarska, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Department of Paediatric Oncology Lund
name: Jacek Toporski, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Paediatric HSCT Unit, Istanbul School of Medicine
name: Sema Anak, Prof MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Dep. of Paediatric Haematology-Oncology and HSCT, Akdeniz University School of Medicine
name: Akif Yesilipek, MD Prof
role: PRINCIPAL_INVESTIGATOR
References:
citation: Peters C, Schrauder A, Schrappe M, von Stackelberg A, Stary J, Yaniv I, Gadner H, Klingebiel T; BFM Study Group, the IBFM-Study Group and the Paediatric Disease Working Party of the EBMT. Allogeneic haematopoietic stem cell transplantation in children with acute lymphoblastic leukaemia: the BFM/IBFM/EBMT concepts. Bone Marrow Transplant. 2005 Mar;35 Suppl 1:S9-11. doi: 10.1038/sj.bmt.1704835.
citation: Pulsipher MA, Peters C, Pui CH. High-risk pediatric acute lymphoblastic leukemia: to transplant or not to transplant? Biol Blood Marrow Transplant. 2011 Jan;17(1 Suppl):S137-48. doi: 10.1016/j.bbmt.2010.10.005.
citation: Peters C, Schrappe M, von Stackelberg A, Schrauder A, Bader P, Ebell W, Lang P, Sykora KW, Schrum J, Kremens B, Ehlert K, Albert MH, Meisel R, Matthes-Martin S, Gungor T, Holter W, Strahm B, Gruhn B, Schulz A, Woessmann W, Poetschger U, Zimmermann M, Klingebiel T. Stem-cell transplantation in children with acute lymphoblastic leukemia: A prospective international multicenter trial comparing sibling donors with matched unrelated donors-The ALL-SCT-BFM-2003 trial. J Clin Oncol. 2015 Apr 10;33(11):1265-74. doi: 10.1200/JCO.2014.58.9747. Epub 2015 Mar 9.
citation: Tasian SK, Peters C. Targeted therapy or transplantation for paediatric ABL-class Ph-like acute lymphocytic leukaemia? Lancet Haematol. 2020 Dec;7(12):e858-e859. doi: 10.1016/S2352-3026(20)30369-0. No abstract available.
citation: Balduzzi A, Dalle JH, Wachowiak J, Yaniv I, Yesilipek A, Sedlacek P, Bierings M, Ifversen M, Sufliarska S, Kalwak K, Lankester A, Toporski J, Di Maio L, Glogova E, Poetschger U, Peters C. Transplantation in Children and Adolescents with Acute Lymphoblastic Leukemia from a Matched Donor versus an HLA-Identical Sibling: Is the Outcome Comparable? Results from the International BFM ALL SCT 2007 Study. Biol Blood Marrow Transplant. 2019 Nov;25(11):2197-2210. doi: 10.1016/j.bbmt.2019.07.011. Epub 2019 Jul 15.