search
Back to results

ALL-SCT BFM International- HSCT in Children and Adolescents With ALL (ALL-SCT-BFMi)

Primary Purpose

Lymphoblastic Leukemia, Acute, Childhood;

Status
Unknown status
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
VP16
TBI
VP16, ATG
TBI
Fludarabine, OKT3, Treosulfan, Thiotepa
VP16, ATG
TBI
Sponsored by
St. Anna Kinderkrebsforschung
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Lymphoblastic Leukemia, Acute, Childhood; focused on measuring ALL, HSCT, children, adolescents

Eligibility Criteria

3 Months - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Universitätsklinik für Kinder- und Jugendheilkunde, Klin. Abt. f. Hämato-Onkologie
  • Universitätsklinik für Kinder- und Jugendheilkunde
  • St. Anna Children's Hospital
  • Department of Paediatric Haematology and Oncology HSCT-Unit
  • Pediatric Clinic II, Rigshospitalet
  • Pediatric Immuno-Hematology Unit Robert Debré Hospital
  • Rambam Medical Center
  • Schneider Children's Medical Center of Israel
  • Clinica Pediatrica dell'Universita di Milano Bicocca, Hospitale San Gerardo
  • Leiden University Hospital
  • Radboud University - Nijmegen Medical Centre
  • Department of Paediatric Haematology and Oncology, Wilhelmina Children's Hospital
  • University Hospital, Collegium Medicum UMK, Pediatric Hematology and Oncology
  • Department of Transplantation, University Children's Hospital
  • Children's University Hospital - Hematology - Oncology
  • Department of Pediadric Oncology, Hematology and Transplantology, University of Medical Sciences
  • Wroclaw Medical University, Dept. of Children Hematology and Oncology
  • Department of Pediatric Bone Marrow Transplantation Unit, University Childrens´ Hospital
  • Department of Pediatric Oncology, Lund University Hospital
  • Department of Pediatrics, Gülhane Military Medical Academy
  • Dept. of Paediatrics - BMT Unit, School of Medicine, University of Ankara
  • Department of Pediatric Hematology-Oncology and Pediatric Stem Cell Transplantation, Akdeniz University School of Medicine
  • Department of Pediatric Hematology, Oncology and BMT, Istanbul School of Medicine
  • Pediatric BMT Centre, Ege University

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Other

Other

Other

Arm Label

MSD - Matched Sibling Donor

MD - Matched Donor

MMD - Mismatched Donor

Arm Description

patients with a MSD receive a conditioning of TBI (12 Gy, 6 fractions) and VP16 60mg/kg for one day (-3)

patients with a HLA matched unrelated Donor (9/10 oder 10/10) receive TBI (12Gy in 6 fractions), VP16 60mg/kg/d on day -3 and ATG fresenius 20mg/kg/d on day -3,-2,-1

Patients with a MMD receive stem cells either from cord blood, a haploidentical donor (parent) or from a non-related donor with a match less or equal 8/10

Outcomes

Primary Outcome Measures

Event free survival
Event-free and overall survival after allogeneic HSCT

Secondary Outcome Measures

number of patients with GvHD acute and chronic Graft-versus-Host-Disease (GvHD)
evaluation of the incidence and severity of acute Grade I-IV graft versus Host disease and of limited or extensive chronic graft versus host disease
occurrence and course of late effects after chemotherapy with subsequent allogeneic HSCT
evaluation of organ dysfunctions according to WHO Toxicity score
occurrence and course of late effects after chemotherapy with subsequent allogeneic HSCT
evaluation of growth retardation and endocrine dysfunction
occurrence and course of late effects after chemotherapy with subsequent allogeneic HSCT
Evaluation of incidence of aseptic bone necrosis.
occurrence and course of subsequent malignancies after chemotherapy with subsequent allogeneic HSCT
Evaluation of incidence of secondary cancer after total body irradiation and/or chemotherapy

Full Information

First Posted
August 23, 2011
Last Updated
June 25, 2015
Sponsor
St. Anna Kinderkrebsforschung
search

1. Study Identification

Unique Protocol Identification Number
NCT01423500
Brief Title
ALL-SCT BFM International- HSCT in Children and Adolescents With ALL
Acronym
ALL-SCT-BFMi
Official Title
Allogeneic Stem Cell Transplantation in Children and Adolescents With Acute Lymphoblastic Leukaemia
Study Type
Interventional

2. Study Status

Record Verification Date
June 2015
Overall Recruitment Status
Unknown status
Study Start Date
January 2007 (undefined)
Primary Completion Date
September 2011 (Actual)
Study Completion Date
September 2016 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
St. Anna Kinderkrebsforschung

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Lymphoblastic Leukemia, Acute, Childhood;
Keywords
ALL, HSCT, children, adolescents

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
405 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
MSD - Matched Sibling Donor
Arm Type
Other
Arm Description
patients with a MSD receive a conditioning of TBI (12 Gy, 6 fractions) and VP16 60mg/kg for one day (-3)
Arm Title
MD - Matched Donor
Arm Type
Other
Arm Description
patients with a HLA matched unrelated Donor (9/10 oder 10/10) receive TBI (12Gy in 6 fractions), VP16 60mg/kg/d on day -3 and ATG fresenius 20mg/kg/d on day -3,-2,-1
Arm Title
MMD - Mismatched Donor
Arm Type
Other
Arm Description
Patients with a MMD receive stem cells either from cord blood, a haploidentical donor (parent) or from a non-related donor with a match less or equal 8/10
Intervention Type
Drug
Intervention Name(s)
VP16
Other Intervention Name(s)
Etoposid
Intervention Description
patients with MSD receive as conditioning VP16 60mg/kg/d on day -3
Intervention Type
Radiation
Intervention Name(s)
TBI
Other Intervention Name(s)
Total body irradiation
Intervention Description
patients with a MSD receive TBI (12Gy in 6 fractions) as conditioning
Intervention Type
Drug
Intervention Name(s)
VP16, ATG
Other Intervention Name(s)
Etoposid, Antithymoglobuline
Intervention Description
patients with a HLA matched unrelated Donor (9/10 oder 10/10) receive VP16 60mg/kg/d on day -3 and ATG fresenius 20mg/kg/d on day -3,-2,-1
Intervention Type
Radiation
Intervention Name(s)
TBI
Other Intervention Name(s)
total body irradiation
Intervention Description
patients with a HLA matched unrelated Donor (9/10 oder 10/10) receive TBI (12Gy in 6 fractions)
Intervention Type
Drug
Intervention Name(s)
Fludarabine, OKT3, Treosulfan, Thiotepa
Other Intervention Name(s)
ATG: Antithymoglobuline
Intervention Description
patients with a MMD (haploidentical or cord blood) receive Fludarabine 30mg/m²/d on day -9 to -5, ATG fresenius 20mg/kg/d on day -3,-2,-1, Treosulfan 14g/m²/d on day -7 to -5 and Thiotepa 2x5mg/kg/d on day -4
Intervention Type
Drug
Intervention Name(s)
VP16, ATG
Other Intervention Name(s)
VP16: Etoposid, ATG: Antithymoglobuline
Intervention Description
patients with MMD-transplantation (8/10)receive VP16 60mg/kg/d on day -4, ATG from day -3 to day-1 20mg/kg/d
Intervention Type
Radiation
Intervention Name(s)
TBI
Other Intervention Name(s)
TBI: total body irradiation
Intervention Description
patients with a MMD-transplantation (8/10) receive 12 Gy in 6 fractions
Primary Outcome Measure Information:
Title
Event free survival
Description
Event-free and overall survival after allogeneic HSCT
Time Frame
10 years
Secondary Outcome Measure Information:
Title
number of patients with GvHD acute and chronic Graft-versus-Host-Disease (GvHD)
Description
evaluation of the incidence and severity of acute Grade I-IV graft versus Host disease and of limited or extensive chronic graft versus host disease
Time Frame
10 years
Title
occurrence and course of late effects after chemotherapy with subsequent allogeneic HSCT
Description
evaluation of organ dysfunctions according to WHO Toxicity score
Time Frame
10 years
Title
occurrence and course of late effects after chemotherapy with subsequent allogeneic HSCT
Description
evaluation of growth retardation and endocrine dysfunction
Time Frame
10 years
Title
occurrence and course of late effects after chemotherapy with subsequent allogeneic HSCT
Description
Evaluation of incidence of aseptic bone necrosis.
Time Frame
10 years
Title
occurrence and course of subsequent malignancies after chemotherapy with subsequent allogeneic HSCT
Description
Evaluation of incidence of secondary cancer after total body irradiation and/or chemotherapy
Time Frame
10 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Months
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Christina Peters Peters, Prof MD PHD
Organizational Affiliation
St. Anna Kinderkrebsforschung
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
Petr Sedlacek, Prof. MD
Organizational Affiliation
Department of Paediatric Haematology and Oncology. HSCT Unit Prague
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Marianne Ifversen, MD
Organizational Affiliation
Paediatric Clinic II, Rigshospitalet Copenhagen
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Jean-Hugues Dalle, Prof. MD
Organizational Affiliation
HSCT Unit Robert Debré Hospital Paris
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Jerry Stein, Prof. MD
Organizational Affiliation
Schneider Children's Medical Center, Israel
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Adriana Balduzzi, MD
Organizational Affiliation
Ospedale San Gerardo Monza
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Marc Bierings, MD
Organizational Affiliation
Wilhelmina Children's Hospital Utrecht
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Jacek Wachowiak, MD, Prof.
Organizational Affiliation
Department of Paediatric Oncology, Haematology and Transplantology, University of Medical Sciences Poznan
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Sabina Sufliarska, MD
Organizational Affiliation
HSCT Unit, University Children's Hospital Bratislava
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Jacek Toporski, MD
Organizational Affiliation
Department of Paediatric Oncology Lund
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Sema Anak, Prof MD
Organizational Affiliation
Paediatric HSCT Unit, Istanbul School of Medicine
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Akif Yesilipek, MD Prof
Organizational Affiliation
Dep. of Paediatric Haematology-Oncology and HSCT, Akdeniz University School of Medicine
Official's Role
Principal Investigator
Facility Information:
Facility Name
Universitätsklinik für Kinder- und Jugendheilkunde, Klin. Abt. f. Hämato-Onkologie
City
Graz
ZIP/Postal Code
8036
Country
Austria
Facility Name
Universitätsklinik für Kinder- und Jugendheilkunde
City
Innsbruck
ZIP/Postal Code
6020
Country
Austria
Facility Name
St. Anna Children's Hospital
City
Vienna
ZIP/Postal Code
A-1090
Country
Austria
Facility Name
Department of Paediatric Haematology and Oncology HSCT-Unit
City
Prague
ZIP/Postal Code
15006
Country
Czech Republic
Facility Name
Pediatric Clinic II, Rigshospitalet
City
Copenhagen
ZIP/Postal Code
4074
Country
Denmark
Facility Name
Pediatric Immuno-Hematology Unit Robert Debré Hospital
City
Paris
ZIP/Postal Code
75935
Country
France
Facility Name
Rambam Medical Center
City
Haifa
ZIP/Postal Code
31096
Country
Israel
Facility Name
Schneider Children's Medical Center of Israel
City
Petach-Tikva
ZIP/Postal Code
49202
Country
Israel
Facility Name
Clinica Pediatrica dell'Universita di Milano Bicocca, Hospitale San Gerardo
City
Monza
ZIP/Postal Code
20052
Country
Italy
Facility Name
Leiden University Hospital
City
Leiden
ZIP/Postal Code
2300
Country
Netherlands
Facility Name
Radboud University - Nijmegen Medical Centre
City
Nijmegen
ZIP/Postal Code
6500
Country
Netherlands
Facility Name
Department of Paediatric Haematology and Oncology, Wilhelmina Children's Hospital
City
Utrecht
ZIP/Postal Code
3584
Country
Netherlands
Facility Name
University Hospital, Collegium Medicum UMK, Pediatric Hematology and Oncology
City
Bydgoszcz
ZIP/Postal Code
85-094
Country
Poland
Facility Name
Department of Transplantation, University Children's Hospital
City
Cracow
ZIP/Postal Code
30-663
Country
Poland
Facility Name
Children's University Hospital - Hematology - Oncology
City
Lublin
ZIP/Postal Code
20-093
Country
Poland
Facility Name
Department of Pediadric Oncology, Hematology and Transplantology, University of Medical Sciences
City
Poznan
ZIP/Postal Code
60-572
Country
Poland
Facility Name
Wroclaw Medical University, Dept. of Children Hematology and Oncology
City
Wroclaw
ZIP/Postal Code
50-345
Country
Poland
Facility Name
Department of Pediatric Bone Marrow Transplantation Unit, University Childrens´ Hospital
City
Bratislava
ZIP/Postal Code
833 40
Country
Slovakia
Facility Name
Department of Pediatric Oncology, Lund University Hospital
City
Lund
ZIP/Postal Code
221-85
Country
Sweden
Facility Name
Department of Pediatrics, Gülhane Military Medical Academy
City
Ankara
ZIP/Postal Code
06018
Country
Turkey
Facility Name
Dept. of Paediatrics - BMT Unit, School of Medicine, University of Ankara
City
Ankara
ZIP/Postal Code
06100
Country
Turkey
Facility Name
Department of Pediatric Hematology-Oncology and Pediatric Stem Cell Transplantation, Akdeniz University School of Medicine
City
Antalya
ZIP/Postal Code
07070
Country
Turkey
Facility Name
Department of Pediatric Hematology, Oncology and BMT, Istanbul School of Medicine
City
Istanbul
ZIP/Postal Code
343990
Country
Turkey
Facility Name
Pediatric BMT Centre, Ege University
City
Izmir
ZIP/Postal Code
35100
Country
Turkey

12. IPD Sharing Statement

Citations:
PubMed Identifier
15812540
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.
Results Reference
result
PubMed Identifier
21195303
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.
Results Reference
result
PubMed Identifier
25753432
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.
Results Reference
result
PubMed Identifier
33242441
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.
Results Reference
derived
PubMed Identifier
31319153
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.
Results Reference
derived

Learn more about this trial

ALL-SCT BFM International- HSCT in Children and Adolescents With ALL

We'll reach out to this number within 24 hrs