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Hematopoietic/Lymphoid CancerPhase 2NCT01982682

Allogeneic Hematopoietic Stem Cell Transplantation for High-Risk Hematologic Malignancies Using One Haploidentical Donor

A Phase 2 study testing Total-Body Irradiation (TBI), Donor Lymphocyte Infusion (DLI) and Cyclophosphamide in people with Hematopoietic/Lymphoid Cancer, 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 total-body irradiation, donor lymphocyte infusion, and cyclophosphamide before donor stem cell transplant works in treating patients with high-risk hematologic malignancies. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Hematopoietic/Lymphoid Cancer
What is being tested?
Total-Body Irradiation (TBI), Donor Lymphocyte Infusion (DLI) and Cyclophosphamide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
41 participants
Where
1 siteUnited States
Study dates
4 Nov 2013to 27 Mar 2017
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

  • This treatment is for patients with high risk hematologic malignancies. High risk is defined as:
  • Any patient with a hematologic malignancy with residual disease after treatment with 1 or more chemotherapy regimens in whom achievement of remission with additional chemoradiotherapy is felt to be unlikely
  • Patients without morphologic evidence of disease but with high risk features which would predict for relapse despite remission at HSCT such as adverse cytogenetics, 3rd or greater CR (complete response), or failure to recover peripheral blood counts to normal ranges. While these patients do not have detectable disease by current methods, like all patients they have non-detectable disease which in their case is highly aggressive.
  • Patients must have one related donor who is HLA (human leukocyte antigen) mismatched in the GVHD direction at two or more HLA loci
  • Patients must adequate organ function:
  • LVEF (left ventricular ejection fraction) of >50 %
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  • Diffusing capacity of the lungs for carbon monoxide (DLCO) (adjusted for hemoglobin) >50 % of predicted and forced expiration to the full FEV-1 >50 %
  • Adequate liver function as defined by a serum bilirubin <1.8, AST (aspartate aminotransferase) or ALT (alanine aminotransferase) < 2.5X upper limit of normal
  • Creatinine clearance of > 60 ml/min
  • Karnofsky Performance Status (KPS) of > 80% on the modified (KPS) tool
  • Patients must be willing to use contraception if they have childbearing potential
  • Able to give informed consent

Who cannot join

  • Modified (KPS) Karnofsky Performance status of <80%
  • > 5 Comorbidity Points on the Hematopoietic cell transplantation - specific comorbidity (HCT-CI) Index (See Appendix B)
  • Class I or II antibodies against donor human leukocyte antigens (HLA)
  • HIV positive
  • Active involvement of the central nervous system with malignancy
  • Psychiatric disorder that would preclude patients from signing an informed consent
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  • Pregnancy, or unwillingness to use contraception if they have child bearing potential
  • Patients with life expectancy of < 6 months for reasons other than their underlying hematologic/oncologic disorder
  • Alemtuzumab treatment within 8 weeks of HSCT admission
  • Anti-thymocyte globulin (ATG) level of > 2 ugm/ml
  • Patients with active inflammatory processes including T max >101 or active tissue inflammation are excluded
  • Inability to tolerate cyclophosphamide or undergo total body irradiation at the doses specified in the treatment plan

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 Allogeneic Hematopoietic Stem Cell Transplantation for High-Risk Hematologic Malignancies Using One Haploidentical Donor

This phase II trial studies how well total-body irradiation, donor lymphocyte infusion, and cyclophosphamide before donor stem cell transplant works in treating patients with high-risk hematologic malignancies. Giving total-body irradiation, donor lymphocyte infusion, and chemotherapy before a donor stem cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. When certain 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Removing the T cells from the donor cells before transplant and giving tacrolimus and mycophenolate mofetil may stop this from happening.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To assess 1 year relapse free survival in high risk patients undergoing hematopoietic stem cell transplant (HSCT) using the Thomas Jefferson University (TJU) 2 step approach with 2 days inserted between the last fraction of total-body irradiation (TBI) and the infusion of donor T cells (donor lymphocyte infusion [DLI]).

SECONDARY OBJECTIVES:

  1. To assess regimen related toxicity in this updated conditioning regimen, graft-versus-host disease (GVHD) incidence and severity, and overall survival in patients undergoing treatment on this protocol.
  2. To assess the consistency and pace of engraftment.
  3. To assess the pace of T cell and B cell immune recovery.

OUTLINE:

CONDITIONING REGIMEN: Patients undergo TBI twice daily (BID) on days -10 to -8, undergo DLI on day -6, and receive cyclophosphamide intravenously (IV) over 2 hours on days -3 and -2.

TRANSPLANT: Patients undergo cluster of differentiation (CD) 34+ selected allogeneic HSCT on day 0.

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

After completion of study treatment, patients are followed up for 1 year.

Being tested
Total-Body Irradiation (TBI), Donor Lymphocyte Infusion (DLI), Cyclophosphamide, Allogeneic hematopoietic stem cell transplantation (HSCT), Mycophenolate mofetil
Medical subject headings
Hematologic Neoplasms

What participants receive

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

  • Total-Body Irradiation (TBI)Radiation

    Undergo TBI

  • Donor Lymphocyte Infusion (DLI)Biological

    Undergo DLI

  • CyclophosphamideDrug

    Also called Endoxan, Cytoxan, Neosar, Procytox

    Given IV

  • Allogeneic hematopoietic stem cell transplantation (HSCT)Procedure or surgery

    Undergo CD34+ selected allogeneic HSCT

  • Mycophenolate mofetilDrug

    Also called CellCept

    Given IV

Study groups in NCT01982682
Study groupRoleWhat they receive
Treatment (TBI, DLI, cyclophosphamide, CD34+ donor HSCT)ExperimentalCONDITIONING REGIMEN: Patients undergo TBI BID on days -10 to -8, undergo DLI on day -6, and receive cyclophosphamide IV over 2 hours on days -3 and -2. TRANSPLANT: Patients undergo CD34+ (cluster of differentiation 34+) selected allogeneic HSCT on day 0. GVHD PROPHYLAXIS: Patients receive tacrolimus IV or PO beginning on day -1 with taper beginning by day 42, and mycophenolate mofetil IV BID on days -1 to 28.
Receives: Radiation: Total-Body Irradiation (TBI); Biological: Donor Lymphocyte Infusion (DLI); Drug: Cyclophosphamide; Procedure: Allogeneic hematopoietic stem cell transplantation (HSCT); Drug: Mycophenolate mofetil

What is being measured

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

  1. Count of Participants That Experience 1 Year Relapse Free Survival After Undergoing Hematopoietic Stem Cell Transplantation (HSCT) Using the Thomas Jefferson University 2 Step Approach

    Measured: Up to 1 year after HSCT

Secondary outcomes (4)

  1. Number of Participants With Successful Engraftment

    Will be reported descriptively. Successful engraftment is defined as ANC (absolute neutrophil count, the number of white blood cells (WBCs) that are neutrophils) ≥ 0.5x109/L for at least 30 days and Platelet engraftment > 20,000 with no transfusion x 7 days.

    Measured: Up to 1 year after HSCT

  2. Count of Participants That Experienced Death as a Result of Graft-versus-host Disease (GVHD)

    Graft versus host disease was clinically characterized based on 4 stages of progression for three major body areas, skin, liver, gut. Subjects who experienced life threatening reactions in their skin, liver and gut ultimately experienced functional impairment and expired.

    Life threatening reactions in the Skin were characterized by desquamation (the shedding of the outer layers of skin) and bullae (a bubblelike cavity filled with air or fluid, in particular).

    Life threatening reactions in the Liver were characterized by Bilirubin, > 15 mg/dl Life threatening reactions in the Gut were characterized by Pain +/- ileus (a painful obstruction of the ileum or other part of the intestine.)

    Measured: Up to 1 year after HSCT

  3. Median Pace of T Cell Immune Recovery at 28 Days Post Hematopoietic Stem Cell Transplantation (HSCT)

    An excess amount of residual T-lymphocytes in the Peripheral Blood Stem Cell Collection (PBSC) product may increase the risk of GVHD. The ideal amount of T-cells left in the PBSC product is no greater than 5x104/kg. Every effort will be made to keep T-cell amounts to below this threshold. The median pace of T-cell immune recovery at 28 days will be monitored by collecting cluster of differentiation (CD24) and (CD38).

    Measured: At 28 days post HSCT

  4. Median Pace of T Cell Immune Recovery at 90 Days Post Hematopoietic Stem Cell Transplantation (HSCT)

    An excess amount of residual T-lymphocytes in the Peripheral Blood Stem Cell Collection (PBSC) product may increase the risk of GVHD. The ideal amount of T-cells left in the PBSC product is no greater than 5x104/kg. Every effort will be made to keep T-cell amounts to below this threshold. The median pace of T-cell immune recovery at 90 days will be monitored by collecting cluster of differentiation (CD24) and (CD38).

    Measured: 90 days post HSCT

Timeline

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

  1. First posted13 Nov 2013 (estimated)
  2. Study start4 Nov 2013
  3. Primary completion27 Mar 2017
  4. Study completion27 Mar 2017

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
41 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01982682
Sponsor's protocol ID
13D.352
Other IDs
2013-031, JT 2974
Status last verified
Apr 2025
First submitted
6 Nov 2013
Last updated
30 Apr 2025
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Total-Body Irradiation (TBI), Donor Lymphocyte Infusion (DLI) and Cyclophosphamide in people with Hematopoietic/Lymphoid Cancer, 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 12 inclusion and 12 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 30 Apr 2025. 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 7 Jun 2018). 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?

Count of Participants That Experience 1 Year Relapse Free Survival After Undergoing Hematopoietic Stem Cell Transplantation (HSCT) Using the Thomas Jefferson University 2 Step Approach (time frame: Up to 1 year after HSCT). 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 NCT01982682.

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 30 Apr 2025.

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