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Alkylating Agent

Cord Blood Transplant for Blood Cancers

Phase 2

Recruiting

Led By Maria Cancio, MD

Research Sponsored by Memorial Sloan Kettering Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients with specific organ function and performance status criteria including Karnofsky or Lansky score ≥ 70%, bilirubin ≤ 1.5 mg/dL, ALT ≤ 3 x upper limit of normal, pulmonary function ≥ 50% predicted, left ventricular ejection fraction ≥ 50%, age-adjusted Hematopoietic Cell Transplantation-Comorbidity Index (aaHCT-CI) ≤ 7, and renal function within specified ranges

Patients meeting specific criteria for Acute Lymphoblastic Leukemia (ALL) including complete first remission (CR1) at high risk for relapse, complete second remission (CR2), and primary refractory or relapsed ALL with minimal residual disease (MRD) after antibody therapy and/or CAR-T cell therapy

Must not have

Patients with inadequate performance status/organ function, advanced metabolic disease, active CNS leukemic involvement, indolent NHL or Hodgkin lymphoma with disease progression, myelofibrosis or other malignancy with moderate-severe bone marrow fibrosis, recent autologous or allogeneic stem cell transplant, active and uncontrolled infection at time of transplantation, HIV infection, seropositivity for HTLV-1, pregnancy or breastfeeding, inability to give informed consent or comply with treatment protocol

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial will look at the one-year mortality rate for people with life-threatening blood disorders who can't get a transplant from a matched donor.

Who is the study for?

This trial is for children and young adults up to age 21 with various high-risk blood cancers or non-malignant disorders, who lack a matched donor for transplantation. Participants must have certain types of leukemia or lymphoma in remission, specific metabolic diseases early in their course, adequate heart, lung, liver and kidney function, and no active central nervous system involvement by cancer.

What is being tested?

The study tests the effectiveness of cord blood transplants combined with chemotherapy drugs (Clofarabine, Fludarabine, Busulfan) and immune suppressants (Cyclosporine-A, Mycophenolate Mofetil) on survival without treatment-related mortality after one year. It's a single-arm study meaning all participants receive the same intervention.

What are the potential side effects?

Possible side effects include reactions to the infusion of cord blood cells; suppression of bone marrow leading to low blood counts; infections due to weakened immune system; damage to organs like liver and kidneys from chemotherapy drugs; graft-versus-host disease where donated cells attack patient's body.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My leukemia is in remission but at high risk of returning, or it has returned after treatment.

Select...

I have a high-risk blood disorder or lymphoma not in remission.

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My AML is in early remission or has less than 10% cancer cells in my bone marrow.

Select...

I am 21 or younger and cannot find a suitable donor quickly.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I do not have severe health issues unrelated to my cancer that would prevent me from safely participating.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Treatment related mortality at 1 year after myeloablative cord transplant

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Participants with non-malignant hematologic disordersExperimental Treatment6 Interventions

Patients with non-malignant disorders receive rituximab IV on day -12 and rabbit anti-thymocyte globulin (rATG) over 12 hours on day -12 to -9. Patients then receive clofarabine IV over 2 hours, fludarabine IV over 30 minutes, and busulfan IV over 3 hours on days -5 to -2. Beginning on day -3, patients receive tacrolimus IV or PO and mycophenolate mofetil IV over at least 2 hours. Patients may begin to taper tacrolimus at approximately 6 months post-transplant and mycophenolate mofetil at approximately 60 days post-transplant in the absence of ongoing GVHD requiring systemic immune suppression. TRANSPLANT: Patients undergo CBT on day 0. POST-TRANSPLANT: Beginning on day 7, patients receive filgrastim SC or IV over 15-30 minutes until ANC recovery. Patients also receive rituximab IV on day 30. Additionally, patients undergo blood sample collection, CT and PET on study.

Group II: Participants with malignant hematologic disordersExperimental Treatment6 Interventions

Patients with malignant disorders receive clofarabine intravenously (IV) over 2 hours, fludarabine phosphate (fludarabine) IV over 30 minutes, and busulfan IV over 3 hours on days -5 to -2. Beginning on day -3, patients receive tacrolimus IV or orally (PO) and mycophenolate mofetil IV over at least 2 hours in the absence of unacceptable toxicity. Patients may begin to taper tacrolimus at approximately 3 months post-transplant and mycophenolate mofetil at approximately 60 days post-transplant in the absence of ongoing graft versus host disease (GVHD) requiring systemic immune suppression. TRANSPLANT: Patients undergo cord blood transplantation (CBT) on day 0.\*\*Subgroup will get rATG (day -12 to -10) POST-TRANSPLANT: Beginning on day 7, patients receive filgrastim subcutaneously (SC) or IV over 15-30 minutes until absolute neutrophil count (ANC) recovery. Additionally, patients undergo blood sample collection, computed tomography (CT) and positron emission tomography (PET) on study.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Mycophenolate Mofetil

1997

Completed Phase 4

~2380

Cyclosporine-A

2005

Completed Phase 3

~60