Trial Outcomes & Findings for Busulfan and Cyclophosphamide Followed By ALLO BMT (NCT NCT01685411)

Last Updated: 2021-04-13

Results Overview

The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

Recruitment status

TERMINATED

Study phase

Target enrollment

5 participants

Primary outcome timeframe

2 Years

Results posted on

2021-04-13

Participant Flow

Participant milestones

Participant milestones
Measure	Allogeneic Hematopoietic Stem Cell Transplant Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Overall Study STARTED	5
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Busulfan and Cyclophosphamide Followed By ALLO BMT

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion
Age, Categorical <=18 years	5 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 Years

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Counts of Participants With Disease Free Survival	3 Participants

PRIMARY outcome

Timeframe: 5 Years

The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Count of Participants With Disease Free Survival	1 Participants

PRIMARY outcome

Timeframe: 7 Years

The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Count of Participants With Disease Free Survival	1 Participants

SECONDARY outcome

Timeframe: By Day 42

Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm\^3 (0.5 x 10\^9/L) or greater.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Count of Participants Who Achieved Neutrophil Engraftment	5 Participants

SECONDARY outcome

Timeframe: Day 100

Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Percentage of Participants With Acute Graft-Versus-Host Disease by Grade Grade 2-4	40 percentage of participants Interval 1.0 to 79.0
Percentage of Participants With Acute Graft-Versus-Host Disease by Grade Grade 3-4	20 percentage of participants Interval 0.0 to 51.0

SECONDARY outcome

Timeframe: 6 Months

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Percentage of Participants With Chronic Graft-Versus-Host Disease	0 percentage of participants Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: 1 Year

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Percentage of Participants With Chronic Graft-Versus-Host Disease	0 percentage of participants Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: 6 Months

In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Percentage of Participants With Treatment-Related Toxicity	0 percentage of participants Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: 1 year

In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Percentage of Participants With Treatment-Related Toxicity	0 percentage of participants Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: 1 Year

The return of disease after its apparent recovery/cessation. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Percentage of Participants With Relapse	40 percentage of participants Interval 1.0 to 79.0

SECONDARY outcome

Timeframe: 2 Years

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Percentage of Participants With Relapse	40 percentage of participants Interval 1.0 to 79.0

SECONDARY outcome

Timeframe: Day 42

Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Percentage of Participants With Engraftment Failure	0 percentage of participants Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: 2 Years

Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Number of Participant Who Were Alive at 2 Years Post Transplant	4 Participants

SECONDARY outcome

Timeframe: 5 Years

Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Number of Participant Who Were Alive at 5 Years Post Transplant	3 Participants

SECONDARY outcome

Timeframe: 7 Years

Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive.

Outcome measures

Outcome measures
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 Participants Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Number of Participant Who Were Alive at 7 Years Post Transplant	1 Participants

Adverse Events

Allogeneic Hematopoietic Stem Cell Transplant

Serious events: 0 serious events

Other events: 5 other events

Deaths: 4 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Allogeneic Hematopoietic Stem Cell Transplant n=5 participants at risk Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Respiratory, thoracic and mediastinal disorders Veno-occlusive disease (VOD)	20.0% 1/5 • Number of events 1 • 7 years
Endocrine disorders Adrenal insufficiency	20.0% 1/5 • Number of events 1 • 7 years
Gastrointestinal disorders Ascites	20.0% 1/5 • Number of events 1 • 7 years
Infections and infestations Bacterial infection	80.0% 4/5 • Number of events 26 • 7 years
Nervous system disorders Brain infarction	20.0% 1/5 • Number of events 1 • 7 years
Cardiac disorders Cardiac dysfunction	20.0% 1/5 • Number of events 1 • 7 years
Eye disorders Cataracts	20.0% 1/5 • Number of events 1 • 7 years
Blood and lymphatic system disorders CSA induced hypertension	40.0% 2/5 • Number of events 2 • 7 years
General disorders Engraftment syndrome	20.0% 1/5 • Number of events 1 • 7 years
Infections and infestations Fungal infection	20.0% 1/5 • Number of events 3 • 7 years
Gastrointestinal disorders GI bleeding	20.0% 1/5 • Number of events 1 • 7 years
Metabolism and nutrition disorders Hyperglycemia	20.0% 1/5 • Number of events 1 • 7 years
Respiratory, thoracic and mediastinal disorders Intubation	20.0% 1/5 • Number of events 3 • 7 years
Nervous system disorders Multifactorial delirium	20.0% 1/5 • Number of events 1 • 7 years
Nervous system disorders Neurotoxicity	20.0% 1/5 • Number of events 2 • 7 years
Musculoskeletal and connective tissue disorders Osteopenia	20.0% 1/5 • Number of events 1 • 7 years
Cardiac disorders Pericardial effusion	20.0% 1/5 • Number of events 1 • 7 years
Infections and infestations Pneumonia	60.0% 3/5 • Number of events 6 • 7 years
Renal and urinary disorders Renal failure	20.0% 1/5 • Number of events 2 • 7 years
Respiratory, thoracic and mediastinal disorders Respiratory failure	20.0% 1/5 • Number of events 1 • 7 years
Nervous system disorders Seizure	20.0% 1/5 • Number of events 1 • 7 years
Respiratory, thoracic and mediastinal disorders Shortness of breath	20.0% 1/5 • Number of events 1 • 7 years
Blood and lymphatic system disorders Transplant-associated thrombotic microangiopathy	20.0% 1/5 • Number of events 1 • 7 years
Infections and infestations Viral infection	40.0% 2/5 • Number of events 11 • 7 years

Additional Information

Margaret L. MacMillan, M.D.

Masonic Cancer Center, University of Minnesota

Phone: 612-626-2778

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place