NCT05477589

Studying Conditioning Regimen In Pediatric Transplantation - AML , SCRIPT-AML

A Randomized, Multi-Center Phase III Trial Comparing Two Conditioning Regimens (CloFluBu and BuCyMel) in Children With Acute Myeloid Leukemia Undergoing Allogeneic Stem Cell Transplantation.

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 170 (estimated)
• Sponsor: Vastra Gotaland Region · Other Government
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

It is a randomized phase 3 study comparing two conditioning regimens in children with Acute Myeloid Leukemia, AML, undergoing allogenic stem cell transplantation. The primary aim is to investigate if a conditioning regimen containing one alkylator (Bu) combined with two antimetabolites (Clo and Flu) results in superior 2-year acute grade III to IV-free, chronic non-limited GvHD-free, relapse free survival than a conditioning regimen combining three alkylating agents (BuCyMel)

Detailed description

The study is designed as an open-label randomized phase III, multicenter superiority trial comparing two conditioning regimens CloFluBu and BuCyMel in children with acute myeloid leukemia (AML) with per-protocol indications to allogeneic hematopoietic stem cell transplantation with a myeloablative conditioning. 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

• Acute Myeloid Leukemia (AML) in Remission
• Stem Cell Transplantation

Interventions

Type	Name	Description
DRUG	busulfan, cyclophosphamide and melphalan, BuCyMel	a three alkylator combination of busulfan, cyclophosphamide and melphalan (BuCyMel, standard arm)
DRUG	clofarabine, fludarabine and busulfan, CloFluBu	combination of clofarabine, fludarabine and busulfan in which two alkylators are replaced by antimetabolites (CloFluBu, experimental arm)

Timeline

• Start date: 2022-06-07
• Primary completion: 2029-12-31
• Completion: 2031-12-31
• First posted: 2022-07-28
• Last updated: 2024-12-19

Locations

17 sites across 10 countries: Belgium, Denmark, Finland, Hong Kong, Israel, Lithuania, Netherlands, Norway, Spain, Sweden