NCT02707393

Brief Summary

In children and adolescents with chronic myeloid leukaemia (CML) stem cell transplantation (SCT) may be a valid alternative to the life-long treatment with tyrosinkinase inhibitors (TKI). This trial aims to evaluate the use of a reduced intensity conditioning regimen (RIC), consisting of fludarabine, melphalan and thiotepa in order to minimize transplant related mortality and toxic late effects. Strict post-transplant monitoring and reintroduction of TKI as well as donor lymphocyte infusions (DLI) in case of relevant residual disease are part of the protocol.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Apr 2009

Longer than P75 for phase_2

Geographic Reach
3 countries

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 30, 2009

Completed
6.8 years until next milestone

First Submitted

Initial submission to the registry

March 1, 2016

Completed
13 days until next milestone

First Posted

Study publicly available on registry

March 14, 2016

Completed
4.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2020

Completed
Last Updated

April 15, 2021

Status Verified

April 1, 2021

Enrollment Period

11.6 years

First QC Date

March 1, 2016

Last Update Submit

April 14, 2021

Conditions

Chronic Myeloid Leukemia

Keywords

stem cell transplantationpediatrics

Outcome Measures

Primary Outcomes (1)

  • transplant related mortality

    one year

Secondary Outcomes (2)

  • overall survival

    five years

  • event free survival

    five years

Study Arms (1)

single arm

EXPERIMENTAL

Fludarabine intravenous - daily dose: 40mg/sqm on day -7, -6, -5, -4; Thiotepa intravenous - daily dose: 2 x 5mg/kg on day -3; Melphalan intravenous - daily dose: 140/mg/sqm on day - 2; ATG intravenous - dose according to local standards on day -3, -2, -1; bone marrow or peripheral blood stem cells of an HLA identical sibling or matched unrelated donor on day 0; GvHD propyhlaxis with Mycophenolate Mofetil and Cyclosporine A

Drug: FludarabineDrug: ThiotepaDrug: MelphalanDrug: ATGDrug: Cyclosporine ADrug: Mycophenolate mofetilBiological: bone marrow or peripheral blood stem cells

Interventions

FludarabineDRUG

infusion

Also known as: Fludara
single arm
ThiotepaDRUG

infusion

Also known as: Thioplex, TESPA
single arm
MelphalanDRUG

infusion

Also known as: Alkeran
single arm
ATGDRUG

infusion

Also known as: Thymoglobulin
single arm
Cyclosporine ADRUG

infusion, orally if possible

Also known as: Sandimmune
single arm
Mycophenolate mofetilDRUG

infusion

Also known as: CellCept
single arm
bone marrow or peripheral blood stem cellsBIOLOGICAL

infusion

single arm

Eligibility Criteria

Age1 Year - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • children and adolescents with BCR/ABL positive CML in chronic phase, who are eligible for allogeneic stem cell transplantation, irrespective of the previous treatment strategy
  • availability of a HLA matched sibling donor (MSD), a matched family donor, a matched unrelated donor or a matched unrelated cord blood (MD)
  • informed consent

You may not qualify if:

  • unavailability of MSD or MD
  • patients in accelerated phase or blast crisis
  • pregnancy
  • previous autologous or allogeneic SCT
  • no informed consent

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Universitätsklinik für Kinder- und Jugendheilkunde

Graz, 8036, Austria

Location

St. Anna Kinderspital

Vienna, 1050, Austria

Location

Hospital Motol, Department of Pediatric Hematology and Oncology, BMT Unit

Prague, 15006, Czechia

Location

Clinica Pediatrica

Monza, 20052, Italy

Location

MeSH Terms

Conditions

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Interventions

fludarabinefludarabine phosphateThiotepaMelphalanthymoglobulinCyclosporineMycophenolic Acid

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsMyeloproliferative DisordersBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

PhosphoramidesOrganophosphorus CompoundsOrganic ChemicalsTriethylenephosphoramideAziridinesAzirinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsPhenylalanineAmino Acids, AromaticAmino Acids, CyclicAmino AcidsAmino Acids, Peptides, and ProteinsCyclosporinsPeptides, CyclicMacrocyclic CompoundsPolycyclic CompoundsPeptidesCaproatesAcids, AcyclicCarboxylic AcidsFatty AcidsLipids

Study Officials

  • Susanne Matthes, MD

    St. Anna Kinderspital

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 1, 2016

First Posted

March 14, 2016

Study Start

April 30, 2009

Primary Completion

December 1, 2020

Study Completion

December 1, 2020

Last Updated

April 15, 2021

Record last verified: 2021-04

Locations

IT(1)AU(2)CZ(1)