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NCT00857389

Thiotepa-Clofarabine-Busulfan With Allogeneic Stem Cell Transplant for High Risk Malignancies

Completed Phase 2 Results posted Last updated 7 April 2020
What this trial tests

Phase 2 trial testing Thiotepa in Stem Cell Transplantation in 60 participants. Completed in 9 March 2019.

Timeline
2 March 2009
Primary endpoint
9 March 2019
9 March 2019

Quick facts

Lead sponsorM.D. Anderson Cancer Center
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment60
Start date2 March 2009
Primary completion9 March 2019
Estimated completion9 March 2019
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

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

Who can join

Under 60, any sex, with Stem Cell Transplantation or Leukemia. 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.

Number of Participants With Survival Rate at 100 Days Post-transplant Primary · 100 days post-transplant

The toxicities will be monitored and scored on a daily basis following the methods of Simon R. Practical Bayesian Guideline for Phase IIB Clinical Trials. A Bayesian stopping rule will be used to stop the trial if there is a 90% chance that the true toxicity rate exceeds the target toxicity rate of 0.25.

GroupValue95% CI
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine45
Number of Participants With Disease Free Survival Secondary · Up to 2 years post transplant

Kaplan-Meier product limit method to estimate the disease free survival.

GroupValue95% CI
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine32
Overall Survival Rate Secondary · Up to 3 years post transplant

Overall Survival Rate will be estimated using the Kaplan-Meier method.

GroupValue95% CI
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine320120 – 833
Graft vs Host Disease (GVHD) Secondary · Up to 30 days post transplant

Severity of toxicities graded according to the NCI Common Toxicity Criteria Adverse Effects (CTCAE) v3.0. Standard reporting guidelines followed for adverse events. Safety data summarized by category, severity and frequency.

GroupValue95% CI
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine34
Engraftment Secondary · up to 100 days post transplant

Engraftment is most commonly defined as the first of three consecutive days of achieving a sustained peripheral blood neutrophil count of \>500 × 10\^6/L .

GroupValue95% CI
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine52
Number of Participants With Serious Adverse Events Secondary · up to 30 days post transplant

Severity of toxicities graded according to the NCI Common Toxicity Criteria Adverse Effects (CTCAE) v3.0. Standard reporting guidelines followed for adverse events. Safety data summarized by category, severity and frequency.

GroupValue95% CI
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine52
Relapse Rate of Participants Treated With Thiotepa, Busulfan, and Clofarabine Secondary · Up to 2 years post transplant

Relapse Rate will be estimated using the Kaplan-Meier method.

GroupValue95% CI
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine26

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events that were related to the preparative regimen and stem cell infusion unil 30 days post transplant.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Conditioning Reg- Thiotepa, Busulfan, and Clofarabine
Serious: 52/58 (90%)
Deaths: 16/58

Serious adverse events (18 terms)

ReactionSystemConditioning Reg- Thiotepa…
Febrile NeutropeniaBlood and lymphatic system disorders
RashSkin and subcutaneous tissue disorders
ALT increaseInvestigations
Hemorrhagic CystitisRenal and urinary disorders
VODHepatobiliary disorders
T bilirubin increasedInvestigations
Creatinine IncreasedInvestigations
PneumonitisRespiratory, thoracic and mediastinal disorders
DAH-diffuse alveolar hemorrhageRespiratory, thoracic and mediastinal disorders
Low granulocyteBlood and lymphatic system disorders
ALK increaseInvestigations
Allergic reactionImmune system disorders
ARDS- Acute respiratory Distress SyndromeRespiratory, thoracic and mediastinal disorders
Ejection fraction decreasedCardiac disorders
HydrocephalusNervous system disorders
HeadacheNervous system disorders
Pulmonary edemaRespiratory, thoracic and mediastinal disorders
EncephalopathyNervous system disorders
Other adverse events (48 terms — click to expand)

ReactionSystemConditioning Reg- Thiotepa…
NauseaGastrointestinal disorders
InfectionInfections and infestations
Oral mucositisGastrointestinal disorders
DiarrheaGastrointestinal disorders
RashSkin and subcutaneous tissue disorders
FeverGeneral disorders
Fluid overloadGeneral disorders
ALT increaseInvestigations
Hemorrhagic CystitisRenal and urinary disorders
T bilirubin increasedInvestigations
ALK increasedInvestigations
HeadacheNervous system disorders
Creatinine IncreasedInvestigations
HypertensionVascular disorders
PneumonitisRespiratory, thoracic and mediastinal disorders
Bone PainMusculoskeletal and connective tissue disorders
Dry eyeEye disorders
Gastrointestinal bleedingGastrointestinal disorders
PruritisSkin and subcutaneous tissue disorders
Abodominal PainGastrointestinal disorders
Hand Foot syndromSkin and subcutaneous tissue disorders
NeuropathyNervous system disorders
Secondary graft failureBlood and lymphatic system disorders
SeizureNervous system disorders
AMS- altered mental statusNervous system disorders
Blurred VisionEye disorders
Broncholitis ObliteransRespiratory, thoracic and mediastinal disorders
DizzinessNervous system disorders
Ejection fraction decreasedCardiac disorders
HSCT related microangiopathy (TA-TMA)Blood and lymphatic system disorders
HypotensionVascular disorders
Low plateletInvestigations
Lower GI track obstructionGastrointestinal disorders
BleedingBlood and lymphatic system disorders
CardiomyopathyCardiac disorders
CellulitisSkin and subcutaneous tissue disorders
ConfusionNervous system disorders
Cystitis noninfectiveRenal and urinary disorders
DAH-diffuse alveolar hemorrhageRespiratory, thoracic and mediastinal disorders
DiplopiaNervous system disorders

Most-reported serious reactions: Febrile Neutropenia, Rash, ALT increase, Hemorrhagic Cystitis, VOD, T bilirubin increased, Creatinine Increased, Pneumonitis.

Data from ClinicalTrials.gov NCT00857389 adverse events section.

Sponsor's own description

Any time the words "you," "your," "I," or "me" appear, it is meant to apply to the potential participant. The goal of this clinical research study is to learn if thiotepa, busulfan, and clofarabine, when given before an allogeneic (bone marrow , blood, or cord blood cells) or haploidentical (bone marrow) stem cell transplantation can help to control cancers of the bone marrow and lymph node system. The safety of this treatment will also be studied. This is an investigational study. Thiotepa and clofarabine are FDA approved and commercially available for the treatment of leukemia. Busulfan is FDA approved and commercially available for use in stem cell transplantation. The combination of thiotepa, clofarabine, and busulfan together with a stem cell transplant is investigational. Up to 60 participants will take part in this study. All will be enrolled at M. D. Anderson.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.

Verify or expand the search:

Other trials of Thiotepa

Trials testing the same drug.

Other recruiting trials for Stem Cell Transplantation

Currently open trials in the same condition.

Other M.D. Anderson Cancer Center trials

Trials by the same sponsor.

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Data sources for this page

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