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Hematopoietic and Lymphoid Cell NeoplasmPhase 2NCT03712878

2-Step Approach to Stem Cell Transplant in Treating Participants With Hematological Malignancies

A Phase 2 study testing Total-Body Irradiation, Donor Lymphocyte Infusion and Cyclophosphamide in people with Hematopoietic and Lymphoid Cell Neoplasm, 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 a 2-step approach to stem cell transplant works in treating patients with blood cancers. Giving chemotherapy and total body irradiation before a lymphocyte (white blood cell) and stem cell transplant helps stop the growth of cells in the bone marrow including normal blood-forming… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Hematopoietic and Lymphoid Cell Neoplasm
What is being tested?
Total-Body Irradiation, Donor Lymphocyte Infusion and Cyclophosphamide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
19 Sep 2018to 19 Mar 2025
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

  • Provide signed and dated informed consent form.
  • Have a hematological malignancy or any type of dyscrasia in which allogeneic HSCT is thought to be beneficial.
  • Have a related donor who is no more than a 1-antigen mismatch at the human leukocyte antigen (HLA)-A; B; C; DR loci in the GVHD direction with the patient. (Patients with a syngeneic donor may be treated on this therapeutic approach, but their outcomes will not be part of the statistical aims of the study.
  • LVEF (left ventricular end diastolic function) of >= 45%.
  • DLCO (diffusing capacity of the lung for carbon monoxide) >= 50% of predicted corrected for hemoglobin.
  • FEV-1 (forced expiratory volume at 1 second >= 50% of predicted.
Show all 14Show fewer
  • Serum bilirubin =< 1.8.
  • Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) =< 2.5 x upper limit of normal.
  • Creatinine clearance of >= 60 mL/min.
  • Have a Hematopoietic Cell Transplant Comorbidity Index (HCT-CI) score =< 5 points (patients with greater than 5 points will be allowed 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 HCT-CI 5 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.
  • Have a Karnofsky performance score (KPS) >= 80%.
  • Women of reproductive potential (defined as women under the age of 50 years still menstruating within 2 months of HSCT despite past history of chemotherapy) will be counseled to use highly effective contraception including oral, intramuscular (IM), or patch contraceptives, intrauterine device (IUD), diaphragm, cervical cap, or contraceptive implant. Pharmacological avoidance of pregnancy and suppression of menstruation may be instituted during the HSCT inpatient stay.
  • Men will be asked to abstain from sexual relations during the treatment period of the HSCT stay.
  • DONOR: All donors are selected and screened for their ability to provide adequate infection-free apheresis products for the patient in a manner that does not put the donor at risk for negative consequences.

Who cannot join

  • Be human immunodeficiency virus (HIV) positive.
  • Be pregnant or breastfeeding.
  • Have received alemtuzumab or rabbit antithymocyte globulin (ATG) within 8 weeks or horse ATG within 6 weeks of the transplant admission. This exclusion criterion will be documented by the absence of these drugs 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 without the specific approval of the PI. If the PI disregards this criterion (example of this is localized prostate cancer not yet requiring treatment), the rationale must be documented in the study binder). This exclusion criterion will be documented by the absence of these drugs in the medical record.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107

Other trials in United States

About this study

Official title: A 2 Step Approach to Matched Related Hematopoietic Stem Cell Transplantation for Patients With Hematological Malignancies-5+5 Dosing

This phase II trial studies how well a 2-step approach to stem cell transplant works in treating patients with blood cancers. Giving chemotherapy and total body irradiation before a lymphocyte (white blood cell) and stem cell transplant helps stop the growth of cells in the bone marrow including normal blood-forming cells (stem cells) and cancer cells. By giving the donor cells in two steps, the dose of lymphocytes given can be tightly controlled and they can be made more tolerant to the body. When the healthy lymphocytes and 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 called graft versus host disease. Giving tacrolimus and mycophenolate mofetil may stop this from happening.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To assess whether providing a patient with "5+5" dosing in a 2 step matched related donor hematopoietic stem cell transplantation (HSCT) increases the percentage of patients who achieve full donor chimerism earlier as defined by 98% or greater donor T cell chimerism at 28 days post HSCT (d+28).

SECONDARY OBJECTIVES:

  1. To assess day (d) +90 chimerism in patients receiving "5+5" dosing. II. To assess post HSCT relapse rates in patients receiving "5+5" dosing. III. To assess rates of grade II-IV graft versus host disease (GVHD) in patients receiving "5+5" dosing.

IV. To assess treatment-related mortality (TRM) in patients receiving "5+5" dosing.

EXPLORATORY OBJECTIVES:

  1. To assess whether patients receiving "5+5" dosing have lower rates of cytomegalovirus (CMV) reactivation as compared to patients treated on Thomas Jefferson University (TJU) 08D.85 (1st Generation 2-Step Matched Related Trial).

OUTLINE:

CONDITIONING REGIMEN: Patients undergo total body irradiation (TBI) twice daily (BID) on days -9 to -6.

TRANSPLANT: Patients receive donor lymphocytes intravenously (IV) on day -6 after the last dose of TBI.

CONDITIONING REGIMEN: Patients receive cyclophosphamide IV on days -3 and -2.

TRANSPLANT: Patients undergo hematopoietic stem cell transplantation on day 0.

GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on day 42 in the absence of GVHD, a suspicion of GVHD, or previous history of GVHD requiring a taper delay. Patients also receive mycophenolate mofetil IV BID beginning on day -1 through day 28 in the absence of GVHD.

After completion of study treatment, patients are followed up at days 28, 90, 180, 270, and 365.

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 IrradiationRadiation

    Also called SCT_TBI, TBI, TOTAL BODY IRRADIATION, Whole Body

    Undergo TBI

  • Donor Lymphocyte InfusionProcedure or surgery

    Also called DLI, Donor Leukocyte Infusion

    Given IV

  • CyclophosphamideDrug

    Also called (-)-Cyclophosphamide, 1-bis(2-chloroethyl)-amino-1-oxo-2-aza-5-oxaphosphoridin monohydrate, 2-[bis(2-chloroethyl)amino]tetrahydro-2H-1,3,2-oxazaphosphorine 2-oxide monohydrate, 2-[bis(b-chloroethyl)amino]-1-oxa-3-aza-2-phosphacyclohexane-2-oxide monohydrate

    Given IV

  • Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery

    Also called Allogeneic, Allogeneic Hematopoietic Cell Transplantation, allogeneic stem cell transplantation, HSC

    Undergo HSCT

  • TacrolimusDrug

    Also called 109581-93-3, FK 506, Fujimycin, Hecoria

    Given IV

  • Mycophenolate MofetilDrug

    Also called 115007-34-6, 128794-94-5, Cellcept, MMF

    Given IV

Study groups in NCT03712878
Study groupRoleWhat they receive
Treatment (TBI, DLI, chemotherapy, HSCT, tacrolimus, MMF)ExperimentalDescription CONDITIONING REGIMEN: Participants undergo TBI BID on days -9 to -6. TRANSPLANT: Participants receive donor lymphocytes IV on day -6 after the last dose of TBI. CONDITIONING REGIMEN: Participants receive cyclophosphamide IV on days -3 and -2. TRANSPLANT: Participants undergo hematopoietic stem cell transplantation on day 0. GVHD PROPHYLAXIS: Participants receive tacrolimus IV beginning on day -1 with taper beginning on day 42 in the absence of GVHD, a suspicion of GVHD, or previous history of GVHD requiring a taper delay. Participants also receive mycophenolate mofetil IV BID beginning on day -1 through day 28 in the absence of GVHD.
Receives: Radiation: Total-Body Irradiation; Procedure: Donor Lymphocyte Infusion; Drug: Cyclophosphamide; Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Drug: Tacrolimus; Drug: Mycophenolate Mofetil

What is being measured

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

  1. Percentage of Participants With Donor T Cell Chimerism Values Greater Than or Equal to 98%

    Donor T cell chimerism (defined by 98% or greater donor T cell chimerism) by 28 days post HSCT (d+28). All estimates of rates will be presented with corresponding 95% confidence intervals. For chimerism rates, the method of Atkinson and Brown will be used to allow for the two-stage design.

    Measured: At day +28

Secondary outcomes (4)

  1. Percentage of Participants With Donor T Cell Chimerism Values Greater Than or Equal to 98%

    Will be presented with corresponding 95% confidence intervals. For chimerism rates, the method of Atkinson and Brown will be used to allow for the two-stage design.

    Measured: At day +90

  2. Relapse Rate

    Will be presented with corresponding 95% confidence intervals.

    Measured: At 1 year post-hematopoietic stem cell transplantation (HSCT)

  3. Percentage of Participants With Grades II-IV Graft Versus Host Disease (GVHD)

    Will be presented with corresponding 95% confidence intervals.

    Measured: Within 1 year of HSCT

  4. Rate of Treatment-related Mortality (TRM)

    Will be presented with corresponding 95% confidence intervals.

    Measured: At 1 year 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 posted19 Oct 2018
  2. Study start19 Sep 2018
  3. Primary completion19 Mar 2025
  4. Study completion19 Mar 2025

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
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03712878
Sponsor's protocol ID
18D.419
Other IDs
JT 12822
Status last verified
Jun 2026
First submitted
17 Oct 2018
Last updated
23 Jul 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 Total-Body Irradiation, Donor Lymphocyte Infusion and Cyclophosphamide in people with Hematopoietic and Lymphoid Cell Neoplasm, 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 14 inclusion and 4 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 23 Jul 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 23 Jul 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?

Percentage of Participants With Donor T Cell Chimerism Values Greater Than or Equal to 98% (time frame: At day +28). 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 NCT03712878.

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 23 Jul 2026; AzTrials last refreshed its copy on 7 Nov 2026.

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