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NCT00475020

Allogeneic Stem Cell Transplantation for Myelofibrosis and Myelodysplastic Syndrome

Completed Phase 2 Results posted Last updated 21 May 2019
What this trial tests

Phase 2 trial testing Busulfan in Myelofibrosis in 63 participants. Completed in 4 October 2017.

Timeline
4 January 2006
Primary endpoint
4 October 2017
4 October 2017

Quick facts

Lead sponsorM.D. Anderson Cancer Center
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment63
Start date4 January 2006
Primary completion4 October 2017
Estimated completion4 October 2017
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

M.D. Anderson Cancer Center — full company profile →

Who can join

Under 75, any sex, with Myelofibrosis. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Rate of Non-relapse Mortality at 100 Days Post-transplant Primary · Non-relapse mortality at 100 days post-transplant

To evaluate the safety of Fludarabine/Busulfan as conditioned regimen for allogeneic stem cell transplantation in patients with myelofibrosis/myelodysplastic syndrome at 100 days post-transplant

Infections
GroupValue95% CI
Participants With Myelofibrosis and Myelodysplastic Syndrome1
Organ Failures
GroupValue95% CI
Participants With Myelofibrosis and Myelodysplastic Syndrome2
Sudden death: likely due to ischemic cardiac event
GroupValue95% CI
Participants With Myelofibrosis and Myelodysplastic Syndrome1
Efficacy of This Therapy 3 Years Post-transplant Secondary · Up to 3 years post-transplant

Efficacy Assessed as Number of Participants with Overall Survival, Leukemia Progression, Primary Graft Failure and Complete Hematological Response. Primary graft failure is defined as failure to achieve an ANC \>/= 0.5 x 10 (9)/L for 3 consecutive days and evidence of donor chimerism by Day +28. Complete hematological response is defined by hemoglobin \>/= 120 g/L; or achievement of transfusion independence, with stable Hb \> 110 g/L, for RBC transfusion-dependent participants; Spleen not palpable; platelet count 150 x 10 (9)/L; White blood cell 4 x 10 (9)/L to 10 x 10(9)/L.

Overall survival at 3 years
GroupValue95% CI
Participants With Myelofibrosis and Myelodysplastic Syndrome25
Leukemia progression
GroupValue95% CI
Participants With Myelofibrosis and Myelodysplastic Syndrome27
Primary graft failures
GroupValue95% CI
Participants With Myelofibrosis and Myelodysplastic Syndrome0
Complete hematological remission
GroupValue95% CI
Participants With Myelofibrosis and Myelodysplastic Syndrome44

Adverse events — posted to ClinicalTrials.gov

Time frame: January 2006 to May 2012. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Participants With Myelofibrosis and Myelodysplastic Syndrome
Serious: 18/58 (31%)
Deaths: 8/58

Serious adverse events (11 terms)

ReactionSystemParticipants With Myelofib…
Secondary graft failureBlood and lymphatic system disorders
Elevated transminitisHepatobiliary disorders
Infectious pneumoniaInfections and infestations
ThrombocytopeniaBlood and lymphatic system disorders
GI GvHDGastrointestinal disorders
Veno-occlusive diseaseHepatobiliary disorders
Renal failureRenal and urinary disorders
Skin GvHDSkin and subcutaneous tissue disorders
Liver GvHDBlood and lymphatic system disorders
Hemolytic anemiaBlood and lymphatic system disorders
Septic shockInfections and infestations
Other adverse events (28 terms — click to expand)

ReactionSystemParticipants With Myelofib…
NauseaGastrointestinal disorders
MucositisGastrointestinal disorders
InfectionsInfections and infestations
ATG induced feversInvestigations
Fluid overloadInvestigations
Elevated transminitisHepatobiliary disorders
Neutropenic feversInvestigations
Skin GvHDSkin and subcutaneous tissue disorders
Upper GI GvHDGastrointestinal disorders
DiarrheaGastrointestinal disorders
Infectious pneumoniaInfections and infestations
Renal insufficiencyRenal and urinary disorders
GI GvHDGastrointestinal disorders
Liver GvHDHepatobiliary disorders
HeadachesGeneral disorders
BK virus associated hemorrhagic cystitisInfections and infestations
FeversInvestigations
HypertensionCardiac disorders
Altered mental status changesNervous system disorders
ATG induced shortness of breathRespiratory, thoracic and mediastinal disorders
AscitesHepatobiliary disorders
Hemorrhagic cystitsRenal and urinary disorders
Cytokine release syndromeInvestigations
CellulitisSkin and subcutaneous tissue disorders
Poor graft functionBlood and lymphatic system disorders
ABO incompatibilityBlood and lymphatic system disorders
Veno-occlusive diseaseHepatobiliary disorders
ATG induced skin rashSkin and subcutaneous tissue disorders

Most-reported serious reactions: Secondary graft failure, Elevated transminitis, Infectious pneumonia, Thrombocytopenia, GI GvHD, Veno-occlusive disease, Renal failure, Skin GvHD.

Data from ClinicalTrials.gov NCT00475020 adverse events section.

Sponsor's own description

The goal of this clinical research study is to learn if using a combination of fludarabine, busulfan, and antithymocyte globulin (ATG) can help to control myelofibrosis or myelodysplastic syndrome in patients receiving a bone marrow or blood stem cell transplant. The safety of these drugs will also be studied.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Optimizing the Conditioning Regimen for Hematopoietic Cell Transplant in Myelofibrosis: Long-Term Results of a Prospective Phase II Clinical Trial.
    Popat U, Mehta RS, Bassett R, Kongtim P, et al · · 2020 · cited 14× · PMID 32438043 · DOI 10.1016/j.bbmt.2020.03.020
  2. Transplantation Outcomes of Myelofibrosis with Busulfan and Fludarabine Myeloablative Conditioning.
    Joseph J, Srour SA, Milton DR, Ramdial JL, et al · · 2023 · cited 6× · PMID 37742746 · DOI 10.1016/j.jtct.2023.09.013

Verify or expand the search:

Other trials of Busulfan

Trials testing the same drug.

Other recruiting trials for Myelofibrosis

Currently open trials in the same condition.

Other M.D. Anderson Cancer Center trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00475020.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing