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Adult Acute Lymphoblastic Leukemia in RemissionPhase 2NCT01384513

A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies

A Phase 2 study testing Fludarabine, Busulfan and Total Body Irradiation (TBI) in people with Adult Acute Lymphoblastic Leukemia in Remission and Adult Acute Myeloid Leukemia in Remission, run by Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This phase II trial studies how well reduced intensity donor stem cell transplant works in treating patients with hematologic malignancies. 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… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Fludarabine, Busulfan and Total Body Irradiation (TBI)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
40 participants
Where
1 siteUnited States
Study dates
4 Aug 2011to 16 Nov 2022
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Any patient with hematologic or oncologic diagnosis in which allogeneic HSCT is thought to be beneficial, and in whom front-line therapy has already been applied. Patients treated on this protocol will be without morphological evidence of disease (complete remission or "CR"), or if the patient has evidence of disease, the patient must have had at least a good partial response (PR) to the most recent therapy and the disease must be chemoresponsive.
  • Patients treated on this study will have:
  • Acute leukemia in 1st or 2nd CR
  • MDS (myelodysplastic syndrome), specific subtypes of RA (refractory anemia) or RARS (refractory anemia with ringed sideroblasts) subtypes.
  • Hodgkin or Indolent Non-Hodgkin's lymphoma with chemosensitive disease
  • Myeloma without morphological evidence of disease, or a deep PR to the most recent therapy
Show all 25Show fewer
  • Myeloproliferative disorders with at least a PR to current therapy
  • Aplastic Anemia
  • A hematological or oncological disease (not listed) that meets the criteria reviewed above (in CR or with a good PR).
  • Patients must have a related donor who is HLA mismatched at 2, 3, or 4 antigens at the HLA-A; B; C; DR loci in the GVHD direction. (Patients with related donors who are HLA identical or are a 1-antigen mismatch may be treated on this therapeutic approach, but will have their outcomes will not be part of the statistical aims of the study (see Summary section).
  • Patients must adequate organ function:
  • LVEF (Left ventricular end diastolic function) of >50%
  • DLCO (Diffusing Capacity of the Lung for Carbon Monoxide ) ≥50% of predicted corrected for hemoglobin
  • Adequate liver function as defined by a serum bilirubin <1.8, AST or ALT < 2.5X upper limit of normal
  • Creatinine Clearance of ≥ 60 mL/min
  • Performance status ≥ 80% (TJU Karnofsky) for patients ≥ 60 years old or ≥70% for patients < 60 years old.
  • HCT-CI Score ≤ 4 points for patients ≥ 60 years old or ≤ 5 points for patients < 60 years old.
  • Patients must have adequate Karnofsky performance status (KPS) and hematopoietic cell transplantation comorbidity index (HCT-CI) scores:
  • Patients < age 60 years must have a KPS of >= 80% and an HCT-CI score of 5 or less
  • Patients aged 60 to 65 years must have a KPS of >= 80% and an HCT-CI score of 4 or less
  • Patients aged 66 to 69 years must have a KPS of 90% and an HCT-CI score of 3 or less
  • Patients aged 70 years or more must have a KPS of 90% and an HCT-CI score of 2 or less
  • Patients with greater than the allowable HCT-CI points for age can be enrolled for trial with approval of the principal investigator (PI) and at least 1 co-investigator (Co-I) not on the primary care team of the patient; this is an adjustment to account for healthy patients who meet the spirit of this protocol but have histories that result in higher than guideline HCT-CI points; an example is a patient with a solid tumor malignancy in their remote history (adds 3 points to HCT-CI total) where the treatment for the malignancy occurred years to decades before and there has been complete recovery of toxicities
  • Patients must be willing to use contraception if they have childbearing potential
  • Able to give informed consent

Who cannot join

  • Human immunodeficiency virus (HIV) positive
  • Active involvement of the central nervous system with malignancy
  • Inability to obtain informed consent
  • Pregnancy
  • Patients with life expectancy of =< 6 months for reasons other than their underlying hematologic/oncologic disorder
  • Patients who have received alemtuzumab or anti-thymocyte globulin (ATG) within 8 weeks of the transplant admission; (documented by the absence of these agents in the medical record)
  • Patients with evidence of another malignancy, exclusive of a skin cancer that requires only local treatment, should not be enrolled on this protocol

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107

Other trials in United States

About this study

Official title: A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies

This phase II trial studies how well reduced intensity donor stem cell transplant works in treating patients with hematologic malignancies. 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving tacrolimus and mycophenolate mofetil after the transplant may stop this from happening. Once the donated stem cells begin working, the patient's immune system may see the remaining cancer cells as not belonging in the patient's body and destroy them. Giving an infusion of the donor's white blood cells (donor lymphocyte infusion) may boost this effect.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To compare the overall survival (OS) rate at 2 years post treatment using the Jefferson 2 step reduced intensity conditioning (RIC) approach in patients with haploidentical family donors with hematological malignancies in morphological or radiographic remission or with chemosensitive, indolent diseases to historical OS rates in similar populations after RIC matched donor HSCT as reported in the literature.

SECONDARY OBJECTIVES:

  1. To compare the treatment-related mortality (TRM) rate at 2 years for patients treated on this study to the historical TRM rates of patients undergoing RIC matched-sibling HSCT as reported in the literature.

II. To compare the 2 year relapse rates and relapse related mortality of patients with myeloid diseases to that of patients with lymphoid diseases who are treated on this Thomas Jefferson University (TJU) RIC 2 step approach.

III. To determine the incidence and severity of graft-versus-host disease (GVHD) in patients undergoing treated on the TJU RIC 2 step approach.

IV. To evaluate engraftment rates and lymphoid reconstitution in patients treated on the TJU RIC 2 step approach.

  1. To evaluate the incidence of TRM at 100 days in patients treated on the TJU RIC 2 step approach.

OUTLINE:

REDUCED INTENSITY CONDITIONING: Patients receive fludarabine phosphate intravenously (IV) over 60 minutes on days -11 to -8 and bulsufan IV over 3 hours on days -10 to -9. Patients undergo total body irradiation (TBI) on day -6. Patients also receive cyclophosphamide IV over 2 hours on days -3 and -2.

TRANSPLANTATION: Patients undergo donor lymphocyte infusion (DLI) on day -6 and cluster of differentiation (CD)-34+ allogeneic peripheral blood stem cell transplantation (PBSCT) on day 0.

GVHD PROPHYLAXIS: Beginning on day -1, patients receive tacrolimus IV or orally (PO) with taper beginning on day 42. Patients also receive mycophenolate mofetil IV twice daily (BID) on days -1 to 28.

After completion of study treatment, patients are followed up periodically for 2 years.

Being tested
Fludarabine, Busulfan, Total Body Irradiation (TBI), Donor Lymphocyte Infusion (DLI), Cyclophosphamide (CY)
Keywords
Allogeneic HSCT, Hematopoietic stem cell transplantation
Medical subject headings
Congenital Abnormalities, Lymphoma, Extranodal NK-T-Cell, Anemia, Aplastic, Pdgfra-Associated Chronic Eosinophilic Leukemia, Leukemia, Myelomonocytic, Chronic, Leukemia, Neutrophilic, Chronic, Thrombocythemia, Essential, Leukemia, Myelomonocytic, Juvenile

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • FludarabineDrug

    Also called fludarabine phosphate, Fludara

    Given IV

  • BusulfanDrug

    Also called Myleran, Busulfex IV

    Given IV

  • Total Body Irradiation (TBI)Radiation

    Also called radiotherapy

    2 Gy administered as part of the conditioning regimen

  • Donor Lymphocyte Infusion (DLI)Biological

    Also called buffy coat fusion

    Undergo DLI

  • Cyclophosphamide (CY)Drug

    Also called Endoxan, Cytoxan, Neosar, Procytox

    Given IV

  • TacrolimusDrug

    Also called FK-506, Fujimycin

    Given IV or PO

  • Mycophenolate mofetilDrug

    Also called MMF, CellCept

    Given IV

  • Allogeneic hematopoietic stem cell transplantationDevice

    Also called CliniMACS

    Undergo CD34+ allogeneic PBSCT

  • Peripheral blood stem cell transplantation (PBSCT)Procedure or surgery

    Also called PBPC transplantation, PBSC transplantation, peripheral blood progenitor cell transplantation, transplantation

    Undergo CD34+ allogeneic PBSCT

Study groups in NCT01384513
Study groupRoleWhat they receive
Treatment (Allogeneic PBSCT)ExperimentalREDUCED INTENSITY CONDITIONING: Patients receive fludarabine phosphate IV over 60 minutes on days -11 to -8 and busulfan IV over 3 hours on days -10 to -9. Patients undergo TBI on day -6. Patients also receive cyclophosphamide IV over 2 hours on days -3 and -2. TRANSPLANTATION: Patients undergo DLI on day -6 and CD-34+ allogeneic PBSCT on day 0. GVHD PROPHYLAXIS: Beginning on day -1, patients receive tacrolimus IV or PO with taper beginning on day 42. Patients also receive mycophenolate mofetil IV BID on days -1 to 28.
Receives: Drug: Fludarabine; Drug: Busulfan; Radiation: Total Body Irradiation (TBI); Biological: Donor Lymphocyte Infusion (DLI); Drug: Cyclophosphamide (CY); Drug: Tacrolimus; Drug: Mycophenolate mofetil; Device: Allogeneic hematopoietic stem cell transplantation; Procedure: Peripheral blood stem cell transplantation (PBSCT)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Survival (OS) in Patients With Haploidentical Family Donors With Hematological Malignancies in Morphological or Radiographic Remission or With Chemosensitive, Indolent Diseases

    The primary null hypothesis is that 2 year OS rate is at most 35%. This hypothesis will be rejected if the 95% confidence interval for year OS rate computed from the estimated Kaplan-Meier survival curves will be entirely above 0.35.

    Measured: At 2 years

Secondary outcomes (7)

  1. Treatment Related Mortality (TRM)

    To compare the treatment related mortality rate (TRM) for patients treated on this study to the historical TRM rates of patients undergoing reduced intensity conditioning (RIC) matched-sibling hematopoietic stem cell transplant (HSCT) as reported in the literature

    Measured: At 2 years

  2. Number of Participants With Acute G2-4 and/or Chronic Moderate or Severe GVHD

    The estimates of incidence rates will be presented with corresponding 95% confidence intervals using the exact method

    Measured: Assessed up to 2 years

  3. Relapse Rate

    Evaluated by estimating Kaplan-Meier survival curves. From these curves, the 95% confidence interval for 2 year rates will be computed.

    Measured: At 2 years

  4. Engraftment Rates

    Hematopoietic engraftment will be defined as:

    • ANC ≥ 0.5x10e9/L for at least 3 days
    • Platelet engraftment >20,000 with no transfusion x 7 days.

    Measured: 44 weeks

  5. Incidence of Treatment Related Mortality (TRM)

    The estimates of incidence rates will be presented with corresponding 95% confidence intervals using the exact method.

    Measured: At 100 days

  6. Relapse Related Mortality

    Evaluated by estimating Kaplan-Meier survival curves. From these curves, the 95% confidence interval for 2 year rates will be computed.

    Measured: At 2 years

  7. Lymphoid Reconstitution

    To evaluate lymphoid reconstitution in patients treated on the Two Step approach

    Measured: 100 days post-transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Jun 2011 (estimated)
  2. Study start4 Aug 2011
  3. Primary completion13 Nov 2020
  4. Study completion16 Nov 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
40 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01384513
Sponsor's protocol ID
11D.247
Other IDs
2011-31, JT 1795
Status last verified
Mar 2026
First submitted
27 Jun 2011
Last updated
15 Apr 2026
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Fludarabine, Busulfan and Total Body Irradiation (TBI) in people with Adult Acute Lymphoblastic Leukemia in Remission and Adult Acute Myeloid Leukemia in Remission, run by Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 25 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 15 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 Apr 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Survival (OS) in Patients With Haploidentical Family Donors With Hematological Malignancies in Morphological or Radiographic Remission or With Chemosensitive, Indolent Diseases (time frame: At 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01384513.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2026.

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