2-Step Stem Cell Transplant for Blood Cancers

Overseen By Dolores Grosso, CRNP

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Sidney Kimmel Cancer Center at Thomas Jefferson University

Must not be taking: Alemtuzumab, Antithymocyte globulin

Disqualifiers: HIV, Pregnancy, Other malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 28

10 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

USAMA GERGIS, MD

Principal Investigator

Sidney Kimmel Cancer Center at Thomas Jefferson University

Eligibility Criteria

This trial is for patients with blood cancers who have a heart function of at least 45%, lung capacity over half the expected, normal liver function, and kidney clearance above 60 mL/min. They need a closely matched related donor for stem cell transplant and must not be HIV positive or have another active cancer (except certain skin cancers). Women should use effective contraception, and men should abstain from sex during treatment.

Inclusion Criteria

I am a donor able to give safe apheresis products without risking my health.

Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) =< 2.5 x upper limit of normal

Have a Hematopoietic Cell Transplant Comorbidity Index (HCT-CI) score =< 5 points

Exclusion Criteria

Be pregnant or breastfeeding

I have received specific immune treatments within 2 months before my transplant.

I have no other cancers besides skin cancer that needs only local treatment, or I have approval from the study leader.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning Regimen

Participants undergo total body irradiation twice daily on days -9 to -6 and receive cyclophosphamide IV on days -3 and -2

9 days

Transplant

Participants receive donor lymphocytes IV on day -6 and undergo hematopoietic stem cell transplantation on day 0

6 days

GVHD Prophylaxis

Participants receive tacrolimus IV beginning on day -1 with taper beginning on day 42 and mycophenolate mofetil IV BID from day -1 through day 28

42 days

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 year

Visits at days 28, 90, 180, 270, and 365

Treatment Details

Interventions

• Allogeneic Hematopoietic Stem Cell Transplantation
• Cyclophosphamide
• Donor Lymphocyte Infusion
• Mycophenolate Mofetil
• Tacrolimus
• Total-Body Irradiation

Trial Overview The study tests a two-step stem cell transplant process to treat blood cancers. It involves chemotherapy, total body irradiation followed by white blood cell and stem cell transplants from donors in controlled doses to help rebuild healthy bone marrow while trying to prevent graft versus host disease using tacrolimus and mycophenolate mofetil.

Participant Groups

Group I: Treatment (TBI, DLI, chemotherapy, HSCT, tacrolimus, MMF) Experimental Treatment 6 Interventions

Description 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.

Allogeneic Hematopoietic Stem Cell Transplantation is already approved in European Union, United States, Canada, Japan for the following indications:

Approved in European Union as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Approved in United States as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Approved in Canada as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Approved in Japan as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Who Is Running the Clinical Trial?

Sidney Kimmel Cancer Center at Thomas Jefferson University

Lead Sponsor

Trials

164

Recruited

10,900+