Studying Conditioning Regimen In Pediatric Transplantation - AML , SCRIPT-AML
NCT ID: NCT05477589
Last Updated: 2024-12-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
170 participants
INTERVENTIONAL
2022-06-07
2031-12-31
Brief Summary
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Detailed Description
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This study is composed of two parts - an interventional part that includes randomization, and an observational part. The interventional part is a phase III randomized, open label, multicenter parallel group trial comparing two conditioning regimens used in pediatric HCT: a three alkylator combination of busulfan, cyclophosphamide and melphalan (BuCyMel, standard arm) and a combination of clofarabine, fludarabine and busulfan in which two alkylators are replaced by antimetabolites (CloFluBu, experimental arm). The observational part will prospectively register outcome measures of transplantation in patients not fulfilling criteria for participation in the interventional part of the study (due to lack of complete remission, lack of matched sibling or unrelated donor, who were not recruited to a national upfront protocol or who decline participation in randomization) but consenting to registration of the data.
Conditions
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Keywords
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Study Design
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RANDOMIZED
FACTORIAL
TREATMENT
NONE
Study Groups
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BuCyMel
a combination of busulfan, cyclophosphamide and melphalan, conditioning regimen
busulfan, cyclophosphamide and melphalan, BuCyMel
a three alkylator combination of busulfan, cyclophosphamide and melphalan (BuCyMel, standard arm)
CloFluBu
a combination of clofarabine, fludarabine and busulfan conditioning regimen
clofarabine, fludarabine and busulfan, CloFluBu
combination of clofarabine, fludarabine and busulfan in which two alkylators are replaced by antimetabolites (CloFluBu, experimental arm)
Interventions
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busulfan, cyclophosphamide and melphalan, BuCyMel
a three alkylator combination of busulfan, cyclophosphamide and melphalan (BuCyMel, standard arm)
clofarabine, fludarabine and busulfan, CloFluBu
combination of clofarabine, fludarabine and busulfan in which two alkylators are replaced by antimetabolites (CloFluBu, experimental arm)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* HCT is performed in a study participating center
* All women of childbearing potential who have to have a negative pregnancy test within 2 weeks prior to the start of treatment.
* Signed informed consent.
* Any relapsed AML after initial treatment according to a defined international AML protocol. (NOPHO-DBH AML 2012/new protocol), or AML in first remission with transplant indications and treatment according to national AML protocol (NOPHO-DBH AML 2012 or new protocol).
* In hematological remission, defined as:
\< 5 % leukemic blasts confirmed by flow cytometry (in patients with an informative leukemia associated immunophenotype) in a bone marrow sample taken ≤14 days prior to start of conditioning and no evidence of extramedullary disease, including in CNS and no leukemic blasts in the peripheral blood (verified by flow cytometry in case immature cells are detected in the peripheral blood differential).
-Patients must have a related or unrelated donor fulfilling any of the following criteria: HLA 10/10 allelic matched, identical, sibling BM donor or HLA 10/10 or 9/10 allelic matched related/unrelated BM or PBSC donor orHLA 5-6/6 unrelated or 6-7-8/8 unrelated Cord Blood (UCB)
* Diagnosis of acute myeloid leukemia
* Indication for allogeneic stem cell transplantation, as defined by primary treatment protocol or treating physician.
* Age ≤18 years at time of initial AML, age ≤ 21 years at transplantation.
* Signed informed consent to prospectively register follow-up data.
Exclusion Criteria
* Diagnosis of juvenile myelomonocytic leukemia (JMML).
* History of previous malignancy (AML diagnosed as secondary cancer).
* Known diagnosis of Fanconi anemia.
* Prior autologous or allogeneic hematopoietic stem cell transplant.
* Planned prophylactic DLI or other immunotherapeutic interventions after HCT that are not included in the upfront protocol, Planned anti-leukemic medication after HCT that are not included in the upfront protocol
* Known intolerance to any of the chemotherapeutic drugs in the protocol.
* Major organ failure precluding administration of planned chemotherapy.
* Patients with uncontrolled bacterial, viral, or fungal infections (currently taking medication and with progression or no clinical improvement) at time of enrollment.
* Severe concomitant disease that does not allow treatment according to the protocol at the investigator's discretion, e.g. malformation syndromes, cardiac malformations, metabolic disorders, renal impairment (\<30% of normal glomerular filtration rate), severe pulmonary, hepatic or cardiac impairment due to toxicity or infection.
* Karnofsky / Lansky score \< 50%
* Females who are pregnant (positive serum or urine βHCG) or breastfeeding.
* Females of childbearing potential or men who have sexual contact with females of childbearing potential unwilling to use effective forms of birth control or abstinence for one year after transplantation.
* Subjects unwilling or unable to comply with the study procedures.
* Diagnosis of Myelodysplastic syndrome (MDS).
* Diagnosis of Juvenile myelomonocytic leukemia (JMML).
* Age above 21 years at time of transplantation
* No consent is given to prospectively register outcome data
* Prior autologous or allogeneic hematopoietic stem cell transplant.
18 Years
ALL
No
Sponsors
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Vastra Gotaland Region
OTHER_GOV
Responsible Party
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Principal Investigators
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Karin Mellgren, Prof. MD
Role: STUDY_CHAIR
Sahlgrenska University Hospital
Birgitta Versluys, MD, Phd
Role: PRINCIPAL_INVESTIGATOR
Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands
Locations
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L'Hôpital Universitaire des Enfants Reine Fabiola (HUDERF)
Brussels, , Belgium
Cliniques Universitaires Saint-Luc (CUSL)
Brussels, , Belgium
Department of Pediatric Hematology, Oncology and SCT, Ghent University Hospital
Ghent, , Belgium
University Hospital Leuven
Leuven, , Belgium
Centre Hospitalier Régional de la Citadelle (CHR)/CHU Liège
Liège, , Belgium
Paediatric Stem Cell Transplant and Immune Deficiency, Department of Pediatric and Adolescent Medicine, Section 4072, Rigshospitalet University Hospital of Copenhagen
Copenhagen, , Denmark
Division of Hematology, Oncology, and Stem Cell Transplantation, The New Children's Hospital, Helsinki University Hospital
Helsinki, , Finland
Department of Pediatrics and Adolescent Medicine, Hong King Children's Hospital
Hong Kong, , Hong Kong
Schneider Children's Medical Center of Israel
Petah Tikva, , Israel
Vilnius University Hospital Santaros Klinikos Center for Pediatric Oncology and Hematology
Vilnius, , Lithuania
Princess Máxima Center for Pediatric Oncology
Utrecht, , Netherlands
Department of Pediatric Hematology and Oncology, Oslo University HospitalOslo University Hospital
Oslo, , Norway
Stemcelltransplant unit Hospital Niño Jesús
Madrid, , Spain
Queen Silvia Children's Hospital, Sahlgrenska University Hospital
Gothenburg, , Sweden
Barncancercentrum, avdelning 64, Skane University Hospital
Lund, , Sweden
Pediatric Hematology immunology and stem cell transplantation Astrid Lindgren children's Hospital Huddinge K86-88
Stockholm, , Sweden
Childrens department for Blood and tumor diseases Uppsala University Hospital
Uppsala, , Sweden
Countries
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Central Contacts
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Facility Contacts
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Pauline Mazilier, MD, PhD
Role: primary
Yousra Hadrani
Role: backup
Cécile Boulanger, MD, PhD
Role: primary
Victoria Bordon, MD, PhD
Role: primary
Marlies Bekaert
Role: backup
Anne Uyttebroeck, MD, PhD
Role: primary
An Michiels
Role: backup
Evelyne Willems, MD, PhD
Role: primary
Catherine Sondag
Role: backup
Marianne Ifversen, MD, PhD
Role: primary
Luise Thellesen
Role: backup
Samppa Ryhänen, MD, PhD
Role: primary
Anna Blubaum, RN
Role: backup
Daniel Cheuk, MD, PhD
Role: primary
Jerry Stein, MD,PhD
Role: primary
Jelena Rascon, MD, PhD
Role: primary
Renata Blackute
Role: backup
Birgitta Versluys, MD, PhD
Role: primary
Jochen Buechner, MD, PhD
Role: primary
Marta Gonzales Vicent Gonzales Vicent, MD, PhD
Role: primary
José M Fernandez
Role: backup
Karin Mellgren, Prof.MD
Role: primary
MD, PhD
Role: backup
Kees-Jan Pronk, MD, PhD
Role: primary
Yvonne Håkansson, RN
Role: backup
Mikael Sundin, MD, PhD
Role: primary
Gunilla Olofsson, RN
Role: backup
Natalja Jackmann, MD, PhD
Role: primary
Other Identifiers
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2021-003282-36
Identifier Type: -
Identifier Source: org_study_id