Clinical trial NCT01423747: Allogeneic Stem Cell Transplantation in Children and Adolescents With Acute Lymphoblastic Leukaemia
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
Collaborators: name: International BFM Study Group
First posted: 2011-08-26
Enrollment: 400
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-SZT BFM international study group wants

to evaluate whether hematopoietic stem cell transplantation (HSCT) from matched family or unrelated matched donors (MD) is equivalent to the HSCT from matched sibling donors (MSD).

to evaluate the efficacy of haematopoietic stem cell transplantation (HSCT) from mismatched family or unrelated mismatched donors (MMD) as compared to HSCT from matched sibling donor (MSD) and matched donor (MD).

to determine whether therapy has been carried out according to the main haematopoietic stem cell transplantation (HSCT) protocol recommendations. The standardisation of the treatment options during haematopoietic stem cell transplantation (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 haematopoietic stem cell transplantation (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 and overall survival after allogeneic haematopoietic stem cell transplantation (HSCT)
timeFrame: 14 years
Secondary outcomes:
measure: occurrence of acute and chronic Graft-versus-Host-Disease (GvHD)
timeFrame: 14 years
measure: occurrence and course of late effects after chemotherapy with subsequent allogeneic hematopoietic stem cell transplantation (HSCT)
timeFrame: 14 years
measure: occurrence and course of late effects after chemotherapy with subsequent allogeneic hematopoietic stem cell transplantation (HSCT)
timeFrame: 14
measure: occurrence and course of late effects after chemotherapy with subsequent allogeneic hematopoietic stem cell transplantation (HSCT)
timeFrame: 14 years
measure: occurrence and course of secondary malignancies after chemotherapy with subsequent allogeneic hematopoietic stem cell transplantation (HSCT)
timeFrame: 14 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 Kinderspital
city: Berlin
country: Germany
facility: Charité Campus Virchow- Klinikum, Klinikum der Pädiatrie, Onkologie/Hämatologie/KMT
city: Dresden
country: Germany
facility: Klinik und Poliklinik für Kinderheilkunde, Hämatologie, Onkologie
city: Düsseldorf
country: Germany
facility: Universitätsklinikum Düsseldorf, Klinik f. Kinderonkologie, Hämatologie u. Immunologie
city: Erlangen
country: Germany
facility: Klinik für Kinder und Jugendliche der Universität Erlangen-Nürnberg
city: Essen
country: Germany
facility: Universitätsklinikum Essen, Zentrum für Kinderheilkunde, Abt. für Hämatologie/Onkologie
city: Frankfurt am Main
country: Germany
facility: Klinik für Kinderheilkunde III, Hämatologie und Onkologie, Johann Wolfgang Goethe Universität
city: Freiburg
country: Germany
facility: Universitätsklinikum Freiburg, Zentrum für Kinderheilkunde und Jugendmedizin, Klinik IV: Päd. Hämatologie und Onkologie
city: Giessen
country: Germany
facility: Zentrum für Kinderheilkunde, Abt. Hämatologie und Onkologie
city: Halle
country: Germany
facility: Klinkum der Med. Fakultät der Martin-Luther-Universität Halle-Wittenberg, Uni. Klinik un Poliklinik für Kinder- und Jugendmedizin
city: Hamburg
country: Germany
facility: Universitätsklinikum Hamburg-Eppendorf, Kinderklinik, Abt. für Hämatologie und Onkologie
city: Hannover
country: Germany
facility: Med. Hochschule Hannover, Päd. Hämatologie und Onkologie
city: Heidelberg
country: Germany
facility: Universitätskinderklinik, Päd. Hämatologie, Onkologie und Immunologie
city: Idar-Oberstein
country: Germany
facility: Klinik für Knochenmarktransplantation
city: Jena
country: Germany
facility: Klinik für Kinder- und Jugendmedizin
city: Kiel
country: Germany
facility: Universitätsklinikum Kiel, Klinik für Allgemeine Pädiatrie
city: München
country: Germany
facility: Klinikum der Universität München, Dr. von Haunersches Kinderspital, Abt. für Hämatologie / Onkologie
city: München
country: Germany
facility: Städt. Krankenhaus München-Schwabing, Universitätskinderklinik der TU
city: Münster
country: Germany
facility: Universitätsklinikum Münster, Klinik und Poliklinik für Kinderheilkunde, päd. Hämatologie / Onkologie
city: Tübingen
country: Germany
facility: Univ.-Klinik für Kinderheilkunde und Jugendmedizin
city: Ulm
country: Germany
facility: Universitätskinderklinik
city: Würzburg
country: Germany
facility: Universitätsklinik, päd. Onkologie/Stammzelltransplantation
Study officials:
affiliation: ALL-REZ BFM Study Center Berlin Germany
name: Arend v. Stackelberg, MD, PhD
role: STUDY_CHAIR
affiliation: ALL BFM study center Kiel, Germany
name: Martin Schrappe, MD, Prof.
role: STUDY_CHAIR
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: Preuner S, Peters C, Potschger U, Daxberger H, Fritsch G, Geyeregger R, Schrauder A, von Stackelberg A, Schrappe M, Bader P, Ebell W, Eckert C, Lang P, Sykora KW, Schrum J, Kremens B, Ehlert K, Albert MH, Meisel R, Lawitschka A, Mann G, Panzer-Grumayer R, Gungor T, Holter W, Strahm B, Gruhn B, Schulz A, Woessmann W, Lion T. Risk assessment of relapse by lineage-specific monitoring of chimerism in children undergoing allogeneic stem cell transplantation for acute lymphoblastic leukemia. Haematologica. 2016 Jun;101(6):741-6. doi: 10.3324/haematol.2015.135137. Epub 2016 Feb 11.
citation: Bader P, Kreyenberg H, von Stackelberg A, Eckert C, Salzmann-Manrique E, Meisel R, Poetschger U, Stachel D, Schrappe M, Alten J, Schrauder A, Schulz A, Lang P, Muller I, Albert MH, Willasch AM, Klingebiel TE, Peters C. Monitoring of minimal residual disease after allogeneic stem-cell transplantation in relapsed childhood acute lymphoblastic leukemia allows for the identification of impending relapse: results of the ALL-BFM-SCT 2003 trial. J Clin Oncol. 2015 Apr 10;33(11):1275-84. doi: 10.1200/JCO.2014.58.4631. Epub 2015 Jan 20.