A Phase 2 interventional study of Cyclophosphamide and Fludarabine Phosphate in Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission and Acute Lymphoblastic Leukemia, sponsored by Roswell Park Cancer Institute. Terminated at 1 site in United States. Open to participants aged 1 Year to 75 Years. Per ClinicalTrials.gov, last updated 2025-07-04.
Sponsored by Roswell Park Cancer Institute · Phase 2, Interventional, and Treatment
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer. Drugs used in chemotherapy, such as fludarabine phosphate and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient?s immune cells and help destroy any remaining cancer cells.
PRIMARY OBJECTIVES:
I. To evaluate the rate of relapse, defined as recurrence of underlying disease or progression of underlying disease, at 1 year in patients who receive haploidentical peripheral blood stem cells (PBSCs) after reduced intensity conditioning and post-transplant cyclophosphamide and tocilizumab (or tocilizumab alternative).
SECONDARY OBJECTIVES:
I. To evaluate safety including development of acute graft versus host disease (GVHD) and death at 100 days post-transplant, as well as other treatment related toxicities including chronic GVHD, engraftment rate, non-relapse mortality, progression free survival (PFS) at one year, and overall survival (OS) at one year, as compared with historical controls.
TERTIARY OBJECTIVES:
I. Correlative studies will include chimerism analysis by molecular analysis and evaluation of immune reconstitution by cytomegalovirus (CMV) dextramer analysis using flow cytometry.
OUTLINE:
Patients receive fludarabine phosphate intravenously (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 2 hours on days -6 and -5. Patients undergo total body irradiation (TBI) on days -1 and peripheral blood stem cell transplantation (PBSCT) on day 0.
After completion of study treatment, patients are followed up at 30 and 100 days.
2,584 studies on the registry are indexed under Precursor Cell Lymphoblastic Leukemia-Lymphoma; 473 are open to participants now.
This study's enrollment of 31 is below the median of 40 across 1,952 interventional studies indexed under Precursor Cell Lymphoblastic Leukemia-Lymphoma.
Browse Precursor Cell Lymphoblastic Leukemia-Lymphoma studies →Roswell Park Cancer Institute is the lead sponsor of 419 studies on the registry; 65 are open to participants now.
Of its 38 completed or terminated interventional studies of FDA-regulated products, 21 (55%) have results posted.
Compared within Precursor Cell Lymphoblastic Leukemia-Lymphoma, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Leukemia, Myeloid, Accelerated Phase. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Suitable related haploidentical donor identified per transplant service:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Child-Pugh class
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients receive fludarabine phosphate IV over 30 minutes on days -6 to -2 and cyclophosphamide IV over 2 hours on days -6 and -5. Patients undergo TBI on days -1 and PBSCT on day 0.
Drug: Cyclophosphamide · Drug: Fludarabine Phosphate · Other: Laboratory Biomarker Analysis · Procedure: Peripheral Blood Stem Cell Transplantation · Radiation: Total-Body Irradiation
Given IV
Also known as: (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida, Ciclofosfamide, Cicloxal, Clafen, Claphene, CP monohydrate, CTX, CYCLO-cell, Cycloblastin, Cycloblastine, Cyclophospham, Cyclophosphamid monohydrate, Cyclophosphamidum, Cyclophosphan, Cyclophosphane, Cyclophosphanum, Cyclostin, Cyclostine, Cytophosphan, Cytophosphane, Cytoxan, Fosfaseron, Genoxal, Genuxal, Ledoxina, Mitoxan, Neosar, Revimmune, Syklofosfamid, WR- 138719
Given IV
Also known as: 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, Fludara, SH T 586
Correlative studies
Undergo PBSCT
Also known as: PBPC transplantation, PBSCT, Peripheral Blood Progenitor Cell Transplantation, peripheral stem cell support, Peripheral Stem Cell Transplant, peripheral stem cell transplantation
Undergo TBI
Also known as: TOTAL BODY IRRADIATION, Whole-Body Irradiation
Relapse Rate
The number of participants that relapse within 1 year.
Time frame: At 1 year
ANC Engraftment Rate, the Percentage of Participants That Had a Successful Engraftment
Absolute Neutrophil Count Engraftment rate was defined as the number of participants that received an engraftment within 1 year post-transplant and ANC \> 0.5x10\^9/L for three consecutive days, out of the total number of participants. The rate was computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
Time frame: At 1 year post-transplant
Platelet Engraftment Rate, the Percentage of Participants That Had a Successful Engraftment
Platelet Engraftment rate was defined as the number of participants that received an engraftment within 1 year post-transplant and the platelets \>= 20 x 10\^9/L after 7 consecutive days with no platelet transfusions, out of the total number of participants. The rate was computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
Time frame: At 1 year post-transplant
Proportion of Participants With Acute Graft Versus Host Disease (GVHD)
Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
Time frame: At 100 days post-transplant
Proportion of Participants With Chronic Graft Versus Host Disease (GVHD)
Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
Time frame: At 1 year post-transplant
Overall Survival
Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson. Will be obtained using the product-limit based Kaplan-Meier method.
Time frame: up to 5 years and 8 months
Progression Free Survival
Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson. Will be obtained using the product-limit based Kaplan-Meier method.
Time frame: up to 5 years and 8 months
Transplant Related Mortality
Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
Time frame: At 1 year post-transplant
Immune Reconstitution
Will be assessed by bone marrow transplantation SOC immunophenotyping panel and by analysis of cytomegalovirus-specific immunity.
Time frame: Up to 1 year
Lymphoid Chimerism Expressed as a Percentage of Donor Cells
Mean lymphoid chimerism expressed as a percentage of donor cells
Time frame: At 30 days
Myeloid Chimerism Expressed as a Percentage of Donor Cells
Mean myeloid chimerism expressed as a percentage of donor cells
Time frame: At 100 days
| Milestone | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Started | 31 |
| Completed | 29 |
| Not completed | 2 |
| Withdrew: Withdrawal by subject | 1 |
| Withdrew: Lost to follow-up | 1 |
The number of participants that relapse within 1 year.
| Participants | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Relapse Rate | 14 |
Absolute Neutrophil Count Engraftment rate was defined as the number of participants that received an engraftment within 1 year post-transplant and ANC \> 0.5x10\^9/L for three consecutive days, out of the total number of participants. The rate was computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
| percentage of participants | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| ANC Engraftment Rate, the Percentage of Participants That Had a Successful Engraftment | 84 (66.0 to 95.0) |
Platelet Engraftment rate was defined as the number of participants that received an engraftment within 1 year post-transplant and the platelets \>= 20 x 10\^9/L after 7 consecutive days with no platelet transfusions, out of the total number of participants. The rate was computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
| percentage of participants | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Platelet Engraftment Rate, the Percentage of Participants That Had a Successful Engraftment | 77 (59 to 90) |
Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
| proportion of participants | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Proportion of Participants With Acute Graft Versus Host Disease (GVHD) | 0.3226 (0.1668 to 0.5137) |
Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
| proportion of participants | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Proportion of Participants With Chronic Graft Versus Host Disease (GVHD) | 0.1935 (0.0745 to 0.3747) |
Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson. Will be obtained using the product-limit based Kaplan-Meier method.
| months | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Overall Survival | 15.7 (7.1 to NA) |
Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson. Will be obtained using the product-limit based Kaplan-Meier method.
| months | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Progression Free Survival | 15.7 (5.5 to 49.5) |
Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
| proportion of participants | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Transplant Related Mortality | 0.0323 (0.0008 to 0.1670) |
Will be assessed by bone marrow transplantation SOC immunophenotyping panel and by analysis of cytomegalovirus-specific immunity.
Results for this outcome have not been posted.
Mean lymphoid chimerism expressed as a percentage of donor cells
| percentage of donor cells | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Lymphoid Chimerism Expressed as a Percentage of Donor Cells | 99.85 ± 0.46 |
Mean myeloid chimerism expressed as a percentage of donor cells
| percentage of donor cells | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Myeloid Chimerism Expressed as a Percentage of Donor Cells | 99.93 ± 0.22 |
Collected over 5 years and 8.5 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) | 17/31 (54.8%) | 8/31 (25.8%) | 27/31 (87.1%) |
| Event | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Infections and infestations - Other, specifyInfections and infestations | 2/31 |
| SepsisInfections and infestations | 2/31 |
| Febrile neutropeniaBlood and lymphatic system disorders | 1/31 |
| Hepatic failureHepatobiliary disorders | 1/31 |
| Immune system disorders - Other, specifyImmune system disorders | 1/31 |
| Lung infectionInfections and infestations | 1/31 |
| Investigations - Other, specifyInvestigations | 1/31 |
| Platelet count decreasedInvestigations | 1/31 |
| Nervous system disorders - Other, specifyNervous system disorders | 1/31 |
| PneumonitisRespiratory, thoracic and mediastinal disorders | 1/31 |
| Event | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| NauseaGastrointestinal disorders | 18/31 |
| DiarrheaGastrointestinal disorders | 17/31 |
| Febrile neutropeniaBlood and lymphatic system disorders | 14/31 |
| FatigueGeneral disorders | 11/31 |
| VomitingGastrointestinal disorders | 10/31 |
| Edema limbsGeneral disorders | 10/31 |
| AnorexiaMetabolism and nutrition disorders | 8/31 |
| Cytokine release syndromeImmune system disorders | 7/31 |
| Infections and infestations - Other, specifyInfections and infestations | 7/31 |
| Lung infectionInfections and infestations | 6/31 |
All treated and eligible patients
| Age, Categorical(Participants) | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 14 |
| >=65 years | 17 |
| Age, Continuous(years) | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Mean | 63 ± 9 |
| Sex: Female, Male(Participants) | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| Female | 11 |
| Male | 20 |
| Race (NIH/OMB)(Participants) | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 4 |
| White | 25 |
| More than one race | 0 |
| Unknown or Not Reported | 2 |
| Region of Enrollment(participants) | Treatment (Fludarabine, Cyclophosphamide, TBI, PBSCT) |
|---|---|
| United States | 31 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Dec 2017 to primary completion in Aug 2023, 68 months.
Results were first posted in Apr 2024, 7 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Jun 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Cyclophosphamide · Fludarabine phosphate
Precursor Cell Lymphoblastic Leukemia-Lymphoma →
Roswell Park Cancer Institute →