Trial Outcomes & Findings for Fludarabine, Cyclophosphamide, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Hematologic Cancer (NCT NCT00134004)

NCT ID: NCT00134004

Last Updated: 2015-10-06

Results Overview

Percentage of participants who die for any reason other than recurrence of disease.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 210 participants
• Primary outcome timeframe: Cumulative incidence for the entire study, up to 11 years
• Results posted on: 2015-10-06

Participant Flow

Participant milestones

Measure	Mini-haplo BMT Non-myeloablative haploidentical bone marrow transplant with a fludarabine, cyclophosphamide (Cy), TBI (total body irradiation) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.
Overall Study STARTED	210
Overall Study COMPLETED	210
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Fludarabine, Cyclophosphamide, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Hematologic Cancer

Baseline characteristics by cohort

Measure	Mini-haplo BMT n=210 Participants Non-myeloablative haploidentical bone marrow transplant with a fludarabine, cyclophosphamide (Cy), TBI (total body irradiation) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.
Age, Continuous	52 years n=5 Participants
Sex: Female, Male Female	61 Participants n=5 Participants
Sex: Female, Male Male	149 Participants n=5 Participants
Region of Enrollment United States	210 participants n=5 Participants

PRIMARY outcome

Timeframe: Cumulative incidence for the entire study, up to 11 years

Percentage of participants who die for any reason other than recurrence of disease.

Outcome measures

Measure	Mini-haplo BMT n=210 Participants Non-myeloablative haploidentical bone marrow transplant with a fludarabine, cyclophosphamide (Cy), TBI (total body irradiation) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.
Transplant-related Mortality	18 percentage of participants

PRIMARY outcome

Timeframe: Cumulative incidence for the entire study, up to 11 years

Percentage of participants who experience disease relapse.

Outcome measures

Measure	Mini-haplo BMT n=210 Participants Non-myeloablative haploidentical bone marrow transplant with a fludarabine, cyclophosphamide (Cy), TBI (total body irradiation) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.
Relapse Rate	55 percentage of participants

PRIMARY outcome

Timeframe: 2 years

Percentage of participants who do not experience disease relapse, disease progression, or death.

Outcome measures

Measure	Mini-haplo BMT n=210 Participants Non-myeloablative haploidentical bone marrow transplant with a fludarabine, cyclophosphamide (Cy), TBI (total body irradiation) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.
Progression-free Survival	34 percentage of participants

SECONDARY outcome

Timeframe: Cumulative incidence for the entire study, up to 11 years

Percentage of participants who experienced failure to engraft (also called graft failure or graft rejection). Failure to engraft is defined as <5% donor chimerism and absence of relapse or any other reason for that chimerism value. All participants who met this criterion were included in this outcome measure.

Outcome measures

Measure	Mini-haplo BMT n=210 Participants Non-myeloablative haploidentical bone marrow transplant with a fludarabine, cyclophosphamide (Cy), TBI (total body irradiation) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.
Graft Failure Rate	13 percentage of participants

SECONDARY outcome

Timeframe: 1 year

Percentage of study participants who experienced a serious adverse event (SAE) within 1 year of bone marrow transplant. Complete data is provided in the Adverse Event tables.

Outcome measures

Measure	Mini-haplo BMT n=210 Participants Non-myeloablative haploidentical bone marrow transplant with a fludarabine, cyclophosphamide (Cy), TBI (total body irradiation) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.
Hematologic and Non-hematologic Toxicities as Measured by NCI Common Toxicity Criteria for Adverse Events, v 3.0 Weekly Until 1 Year After Transplantation	9.5 percentage of participants

Adverse Events

Mini-haplo BMT

Serious events: 20 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Measure	Mini-haplo BMT n=210 participants at risk Non-myeloablative haploidentical bone marrow transplant with a fludarabine, cyclophosphamide (Cy), TBI (total body irradiation) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.
Cardiac disorders Atrial fibrillation	0.48% 1/210 • Number of events 1
Renal and urinary disorders Acute kidney injury	0.95% 2/210 • Number of events 2
Nervous system disorders Altered mental status	0.48% 1/210 • Number of events 1
Respiratory, thoracic and mediastinal disorders Acute respiratory distress syndrome	0.48% 1/210 • Number of events 1
Gastrointestinal disorders Ascites	0.48% 1/210 • Number of events 1
Gastrointestinal disorders Cholestasis	0.48% 1/210 • Number of events 1
Respiratory, thoracic and mediastinal disorders Dyspnea	0.48% 1/210 • Number of events 1
Infections and infestations Fungemia	1.4% 3/210 • Number of events 3
Gastrointestinal disorders GI bleed	0.95% 2/210 • Number of events 2
Respiratory, thoracic and mediastinal disorders Idiopathic pneumonia syndrome	0.48% 1/210 • Number of events 1
Investigations Multi-organ failure	0.48% 1/210 • Number of events 1
Infections and infestations Neutropenic fever	0.48% 1/210 • Number of events 1
Infections and infestations Pneumonia	2.4% 5/210 • Number of events 5
Nervous system disorders Posterior reversible encephalopathy syndrome	0.48% 1/210 • Number of events 1
Respiratory, thoracic and mediastinal disorders Pulmonary Vascular Obstructive Disease	0.48% 1/210 • Number of events 1
Renal and urinary disorders Renal failure	1.9% 4/210 • Number of events 4
Respiratory, thoracic and mediastinal disorders Respiratory failure	3.8% 8/210 • Number of events 8
Nervous system disorders Seizure	0.48% 1/210 • Number of events 1
Infections and infestations Sepsis	2.9% 6/210 • Number of events 6
Vascular disorders Subdural hematoma	1.4% 3/210 • Number of events 3

Other adverse events

Measure	Mini-haplo BMT n=210 participants at risk Non-myeloablative haploidentical bone marrow transplant with a fludarabine, cyclophosphamide (Cy), TBI (total body irradiation) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.
Infections and infestations Pneumonia	5.2% 11/210 • Number of events 11

Additional Information

Dr. Ephraim Fuchs

Johns Hopkins University

Phone: 410-419-6479

Email: fuchsep@jhmi.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place