Skip to content
Triangle logo TriangleGet started
← Study library

NCT03333486 · REGISTRY CONDITION SEARCH

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase
PHASE2
Status at capture
TERMINATED
Registry last update
2025-07-04

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

→

baseline

Baseline characteristics

→

outcome · POSTED

Relapse Rate

→

outcome · POSTED

ANC Engraftment Rate, the Percentage of Participants That Had a Successful Engraftment

→

outcome · POSTED

Platelet Engraftment Rate, the Percentage of Participants That Had a Successful Engraftment

→

outcome · POSTED

Proportion of Participants With Acute Graft Versus Host Disease (GVHD)

→

outcome · POSTED

Proportion of Participants With Chronic Graft Versus Host Disease (GVHD)

→

outcome · POSTED

Overall Survival

→

outcome · POSTED

Progression Free Survival

→

outcome · POSTED

Transplant Related Mortality

→

outcome · NOT POSTED

Immune Reconstitution

→

outcome · POSTED

Lymphoid Chimerism Expressed as a Percentage of Donor Cells

→

outcome · POSTED

Myeloid Chimerism Expressed as a Percentage of Donor Cells

→

adverse events

Adverse events

→

more info

Limitations, agreements, and source contact

→
Who could take part
eligibility Criteria
Inclusion Criteria: * Any disease that is considered transplant eligible per TCT standards * Disease response noted (i.e. CR, non-CR, or not applicable): Assessed as per disease specific criteria * Suitable related haploidentical donor identified per transplant service: * Recipient should not have HLA antibodies to potential donor. If the recipient does have HLA antibodies to the potential donor, an alternative donor is preferred; however, if there are no suitable alternative donors, the anti-HLAt antibodies should be depleted per transplant service guidelines. * Haploidentical donors that are ABO compatible with the recipient are preferred. Minor ABO incompatibility is preferred to major ABO incompatibility. Major ABO incompatibility between recipient and donor is the least preferred but still acceptable for this study. * It is preferred that the haploidentical donor must be available to donate on day -1 and day 0, so that fresh product can be processed by the Stem Cell lab and administered to the patient on day 0.While less preferable, cryopreserved product may be utilized with this product. * Diffusing capacity of the lung for carbon monoxide (DLCO) \> 40% predicted, corrected for hemoglobin and/or alveolar ventilation * Left ventricular ejection fraction \> 40% * Bilirubin, liver alkaline phosphatase, serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamate pyruvate transaminase (SGPT) =\< 3 x upper limit of normal * Calculated creatinine clearance \> 40 cc/min by the modified Cockcroft-Gault formula for adults or the Schwartz formula for pediatrics * Have a Karnofsky (adult) or Lansky (for =\< 16 years) performance status \>= 60% * Patient must be able to pass radiation evaluation (i.e.: able to receive 200 cGy) * Patients who have failed a prior autologous transplant are eligible; however, at least 90 days must have elapsed between the start of this reduced intensity conditioning regimen and the last transplant if patient had a prior autologous BMT * Participants of child-bearing potential must agree to use adequate contraceptive methods (e.g., hormonal or barrier method of birth control; abstinence) prior to study entry; should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately * Participant must understand the investigational nature of this study and sign an independent ethics committee/Institutional Review Board approved written informed consent form prior to receiving any study related procedure * If patient is planned to use a fully matched donor, patient is excluded from trial; patient must be planned to undergo a haploidentical matched transplant to participate on study. Patient is still eligible for trial regardless of donor options if PI feels that haplo transplant is in the patient's best interest per clinical decision * Exclusion Criteria: * Participants who have had chemotherapy (not including molecularly targeted agents; examples include, but are not limited to, tyrosine kinase inhibitors such as FLT3 inhibitors and IDH2 inhibitors), radiation treatment and/or surgery 7 days prior to starting conditioning regimen. Those who have not recovered sufficiently from adverse events due to agents administered more than 2 weeks earlier are also ineligible. Exceptions may be made on a case-by-case basis after discussion with the PI * Uncontrolled central nervous system (CNS) disease (for hematologic malignancies) Per PI discretion * Child-Pugh class B and C liver failure * Concomitant active malignancy that would be expected to require chemotherapy within 3 years of transplant (other than non-melanoma skin cancer) Exception would include any concurrently existing malignancy that could be treated with a transplant per PI discretion (Example: Patient has AML but a history of mastocytosis) * Patients who have received maximally allowed doses (given in 2 Gy fractionations, or equivalent) of previous radiation therapy to various organs; patients who previously have received a higher than allowed dose of radiation to a small lung, liver and brain volume, will be evaluated by the radiation oncologist to determine if the patient is eligible for study * Uncontrolled diabetes mellitus, cardiovascular disease, active serious infection or other condition which, in the opinion of treating physician, would make this protocol unreasonably hazardous for the patient * Known human immunodeficiency virus (HIV) positive * Pregnant or nursing female participants * Patients who in the opinion of the treating physician are unlikely to comply with the restrictions of allogeneic stem cell transplantation based on formal psychosocial screening * Patients with donor specific HLA antibodies with a titer greater than 3000 MFI (whether or not they have undergone a desensitization protocol) * Patients who have undergone a prior allogeneic hematopoietic or (other organ) transplant * Treating physician considers the potential HLA haploidentical donor to be ineligible to receive G-CSF, and/or concern on the part of the treating physician for risk of harm to the potential donor with administration of G-CSF, and/or refusal by the potential donor (or donor's guardian) to receive G-CSF * Any condition which in the investigator's opinion deems the participant an unsuitable candidate to receive study drug * Received an investigational agent within 14 days prior to enrollment. Exceptions may be made on a case-by-case basis after discussion with PI
healthy Volunteers
false
maximum Age
75 Years
minimum Age
1 Year
sex
ALL
std Ages
  1. CHILD
  2. ADULT
  3. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. description
    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.
    intervention Names
    1. Drug: Cyclophosphamide
    2. Drug: Fludarabine Phosphate
    3. Other: Laboratory Biomarker Analysis
    4. Procedure: Peripheral Blood Stem Cell Transplantation
    5. Radiation: Total-Body Irradiation
    label
    Treatment (fludarabine, cyclophosphamide, TBI, PBSCT)
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Treatment (fludarabine, cyclophosphamide, TBI, PBSCT)
    description
    Given IV
    name
    Cyclophosphamide
    other Names
    1. (-)-Cyclophosphamide
    2. 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate
    3. Carloxan
    4. Ciclofosfamida
    5. Ciclofosfamide
    6. Cicloxal
    7. Clafen
    8. Claphene
    9. CP monohydrate
    10. CTX
    11. CYCLO-cell
    12. Cycloblastin
    type
    DRUG
  2. arm Group Labels
    1. Treatment (fludarabine, cyclophosphamide, TBI, PBSCT)
    description
    Given IV
    name
    Fludarabine Phosphate
    other Names
    1. 2-F-ara-AMP
    2. 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-
    3. Beneflur
    4. Fludara
    5. SH T 586
    type
    DRUG
  3. arm Group Labels
    1. Treatment (fludarabine, cyclophosphamide, TBI, PBSCT)
    description
    Correlative studies
    name
    Laboratory Biomarker Analysis
    type
    OTHER
  4. arm Group Labels
    1. Treatment (fludarabine, cyclophosphamide, TBI, PBSCT)
    description
    Undergo PBSCT
    name
    Peripheral Blood Stem Cell Transplantation
    other Names
    1. PBPC transplantation
    2. PBSCT
    3. Peripheral Blood Progenitor Cell Transplantation
    4. peripheral stem cell support
    5. Peripheral Stem Cell Transplant
    6. peripheral stem cell transplantation
    type
    PROCEDURE
  5. arm Group Labels
    1. Treatment (fludarabine, cyclophosphamide, TBI, PBSCT)
    description
    Undergo TBI
    name
    Total-Body Irradiation
    other Names
    1. TOTAL BODY IRRADIATION
    2. Whole-Body Irradiation
    type
    RADIATION
Study design
allocation
NA
intervention Model
SINGLE_GROUP
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
31
type
ACTUAL
Registered outcome plans (not posted results)
other Outcomes
  1. description
    Will be assessed by bone marrow transplantation SOC immunophenotyping panel and by analysis of cytomegalovirus-specific immunity.
    measure
    Immune Reconstitution
    time Frame
    Up to 1 year
  2. description
    Mean lymphoid chimerism expressed as a percentage of donor cells
    measure
    Lymphoid Chimerism Expressed as a Percentage of Donor Cells
    time Frame
    At 30 days
  3. description
    Mean myeloid chimerism expressed as a percentage of donor cells
    measure
    Myeloid Chimerism Expressed as a Percentage of Donor Cells
    time Frame
    At 100 days
primary Outcomes
  1. description
    The number of participants that relapse within 1 year.
    measure
    Relapse Rate
    time Frame
    At 1 year
secondary Outcomes
  1. description
    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.
    measure
    ANC Engraftment Rate, the Percentage of Participants That Had a Successful Engraftment
    time Frame
    At 1 year post-transplant
  2. description
    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.
    measure
    Platelet Engraftment Rate, the Percentage of Participants That Had a Successful Engraftment
    time Frame
    At 1 year post-transplant
  3. description
    Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
    measure
    Proportion of Participants With Acute Graft Versus Host Disease (GVHD)
    time Frame
    At 100 days post-transplant
  4. description
    Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
    measure
    Proportion of Participants With Chronic Graft Versus Host Disease (GVHD)
    time Frame
    At 1 year post-transplant
  5. description
    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.
    measure
    Overall Survival
    time Frame
    up to 5 years and 8 months
  6. description
    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.
    measure
    Progression Free Survival
    time Frame
    up to 5 years and 8 months
  7. description
    Will be computed with corresponding exact 95% confidence intervals based on the methodology of Clopper and Pearson.
    measure
    Transplant Related Mortality
    time Frame
    At 1 year post-transplant
Full study description
brief Summary
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.
detailed Description
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.
Source references
Source notices and limitations
    Discovery and provenance
    1. release id
      ctgov-registry-1336d216cbd1bbff780dac70
      edge id
      nct-edge-53a246ae95588f434481
      requested nct id
      NCT03333486
      primary nct id
      NCT03333486
      source kind
      ctgov_condition_query
      source record id
      Source null
      source file sha256
      cddcd4e3a59550bc78924062a298c359d8f811413f73f4a4461963aecf3790d6
      payload sha256
      3596dc0414e8666faa934e5245dc475212895f772fb9b83ad69e55cd7eef8e42
      payload
      clinical indication matching status
      condition_index_match_asserted_by_registry_not_reviewed
      end exclusive
      true
      id
      nct-edge-53a246ae95588f434481
      link basis
      returned_by_explicit_condition_and_posted_results_filter_query
      nct id
      NCT03333486
      occurrences
      1. context exact
        NCT03333486
        end
        11
        exact text
        NCT03333486
        start
        0
      offset unit
      unicode_code_points
      query cond
      Juvenile Myelomonocytic Leukemia
      query disease state id
      ds-juvenile-myelomonocytic-leukemia
      source file
      /Users/arunverma/Documents/ChatGPT/Cancer Statistics Website/research-data/2026-09-10/clinicaltrials-condition/round-0000/raw/cond-juvenile-myelomonocytic-leukemia-000.json
      source file sha256
      cddcd4e3a59550bc78924062a298c359d8f811413f73f4a4461963aecf3790d6
      source json pointer
      /studies/70/protocolSection/identificationModule/nctId
      source kind
      ctgov_condition_query
      source record id
      Source null
      source string basis
      parsed_JSON_string_unmodified
      source string sha256
      5cce54b5b737a3395b23b323a710c2ea0c603df07d3f9e2edfd56942c5fe75b3
    2. release id
      ctgov-registry-1336d216cbd1bbff780dac70
      edge id
      nct-edge-724c7115554cd73fff37
      requested nct id
      NCT03333486
      primary nct id
      NCT03333486
      source kind
      ctgov_condition_query
      source record id
      Source null
      source file sha256
      2314ce01316b2d7044d298c6d25eda12e99cc5f44273bc75efed6cd5e03a8c14
      payload sha256
      260e1086e065dbafbfa12c45af82e789de363350331f34ed94732c53495d85ee
      payload
      clinical indication matching status
      condition_index_match_asserted_by_registry_not_reviewed
      end exclusive
      true
      id
      nct-edge-724c7115554cd73fff37
      link basis
      returned_by_explicit_condition_and_posted_results_filter_query
      nct id
      NCT03333486
      occurrences
      1. context exact
        NCT03333486
        end
        11
        exact text
        NCT03333486
        start
        0
      offset unit
      unicode_code_points
      query cond
      Plasma Cell Neoplasms
      query disease state id
      ds-plasma-cell-neoplasms
      source file
      /Users/arunverma/Documents/ChatGPT/Cancer Statistics Website/research-data/2026-09-10/clinicaltrials-condition/round-0000/raw/cond-plasma-cell-neoplasms-000.json
      source file sha256
      2314ce01316b2d7044d298c6d25eda12e99cc5f44273bc75efed6cd5e03a8c14
      source json pointer
      /studies/504/protocolSection/identificationModule/nctId
      source kind
      ctgov_condition_query
      source record id
      Source null
      source string basis
      parsed_JSON_string_unmodified
      source string sha256
      5cce54b5b737a3395b23b323a710c2ea0c603df07d3f9e2edfd56942c5fe75b3
    3. release id
      ctgov-registry-1336d216cbd1bbff780dac70
      edge id
      nct-edge-e8ec9a1bd7917a18c841
      requested nct id
      NCT03333486
      primary nct id
      NCT03333486
      source kind
      ctgov_condition_query
      source record id
      Source null
      source file sha256
      e270600534699a676fe83226e7a7b0f73aeb00c1d0a05cfc115c748864ad8173
      payload sha256
      2f348b3f24c12f12e65c4f2d95071ad801ede852c3607927e802fb24095bdda5
      payload
      clinical indication matching status
      condition_index_match_asserted_by_registry_not_reviewed
      end exclusive
      true
      id
      nct-edge-e8ec9a1bd7917a18c841
      link basis
      returned_by_explicit_condition_and_posted_results_filter_query
      nct id
      NCT03333486
      occurrences
      1. context exact
        NCT03333486
        end
        11
        exact text
        NCT03333486
        start
        0
      offset unit
      unicode_code_points
      query cond
      Peripheral T-Cell Non-Hodgkin Lymphoma
      query disease state id
      ds-peripheral-t-cell-non-hodgkin-lymphoma
      source file
      /Users/arunverma/Documents/ChatGPT/Cancer Statistics Website/research-data/2026-09-10/clinicaltrials-condition/round-0000/raw/cond-peripheral-t-cell-non-hodgkin-lymphoma-000.json
      source file sha256
      e270600534699a676fe83226e7a7b0f73aeb00c1d0a05cfc115c748864ad8173
      source json pointer
      /studies/59/protocolSection/identificationModule/nctId
      source kind
      ctgov_condition_query
      source record id
      Source null
      source string basis
      parsed_JSON_string_unmodified
      source string sha256
      5cce54b5b737a3395b23b323a710c2ea0c603df07d3f9e2edfd56942c5fe75b3

    Preserved source evidence · Not medical advice