Stem Cell Transplant for Blood Cancer

Recruiting at 2 trial locations

Overseen By Miguel-Angel Perales, MD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Memorial Sloan Kettering Cancer Center

Disqualifiers: HIV, Pregnancy, Infection, others

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

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 30

17 Participants Needed

Trial Summary

What is the purpose of this trial?

The main purpose of this study is to learn if a new combination of chemotherapy, in combination with low-dose radiation, will be safe for the patient, and at the same time provide the best opportunity to cure the bone marrow cancer. The combination of chemotherapy and radiation described in the study is considered 'low intensity.' Although the chemotherapy agents used in this study and for transplant are FDA approved, the chemotherapy treatment and conditioning regimens or combinations listed in this consent are not yet FDA approved. The CliniMACS device is FDA approved for one type of T cell depletion (positive selection of the stem cells) but not approved yet for other type of T cell depletion, which is being studied on this protocol. This pilot study, along with other studies will serve as the basis for FDA approval, if outcomes are favorable.

Research Team

Roni Tamari, MD

Principal Investigator

Memorial Sloan Kettering Cancer Center

Eligibility Criteria

This trial is for adults with certain high-risk blood cancers or severe aplastic anemia who haven't had a previous stem cell transplant. They should be responding to current therapy, have adequate organ function, and not be pregnant or HIV positive. A detailed consent form must be signed indicating willingness to participate.

Inclusion Criteria

I am able to care for myself and am up more than 50% of my waking hours.

Patients must be willing to participate as a research participant and sign an informed consent form

My heart, liver, kidneys, and lungs are all working well.

Exclusion Criteria

I have had a stem cell transplant from a donor.

I am HIV positive.

I have tested positive for HTLV-1.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning

Participants undergo transplant conditioning with ATG, fludarabine, and TBI, followed by high dose cyclophosphamide post stem cell infusion

8 days

Inpatient stay

Transplantation

Stem cell infusion and administration of Rituxan to reduce EBV viremia risk

1 day

Inpatient stay

Post-Transplant Monitoring

Participants receive G-CSF and undergo chimerism studies and immune function assessments

24 months

Regular follow-up visits at 30 days, 100 days, 6, 12, 18, and 24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Treatment Details

Interventions

• Allogeneic Hematopoietic Stem Cell Transplantation

Trial Overview The study tests a new low-intensity combination of chemotherapy with low-dose radiation before a T cell depleted allogeneic hematopoietic stem cell transplantation. The goal is to determine safety and effectiveness in curing bone marrow cancer.

Participant Groups

Group I: Patients with Myeloid Malignancies & Aplastic Anemia Experimental Treatment 6 Interventions

Transplant conditioning will consist of: ATG (2 mg/kg/day IV on days-8 through-6), fludarabine (30 mg/m\^2/d on days -5 through -2), TBI 400 cGy in 2 divided doses (days -2 and -1) and high dose cyclophosphamide given post stem cell infusion (50 mg/kg on days +3 and +4). One dose of Rituxan (200 mg/m\^2) will be given to reduce the risk of EBV viremia. The donor stem cell product will be derived from the peripheral blood with a target cell infusion of ≥8X10\^6 CD34 cells per recipient kg. Patients will receive post-transplant G-CSF starting on day +7. Patients will undergo donor/recipient bone marrow and peripheral chimerism studies at 30 and 100, and 6, 12, 18 and 24 months post allo HCT and thereafter, at the discretion of the treating clinician. Immune function and disease restaging will be performed at day 100 and 6, 12, 18, and 24 months and as otherwise clinically indicated by the treating physician.

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?

Memorial Sloan Kettering Cancer Center

Lead Sponsor

Trials

1,998

Recruited

602,000+