Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

Part of paid clinical trials in New York, New York.

Sponsor
Memorial Sloan Kettering Cancer Center
Study ID
NCT04644016
Phase
PHASE2
Status
Recruiting
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Conditions

  • ALL
  • AML
  • Bone Marrow Failure
  • HLH
  • Hemoglobinopathies
  • Inherited Metabolic Disorders
  • MDS
  • MPD Withou Myelofibrosis
  • NHL or HL

Eligibility Criteria

Sex
ALL
Age
N/A - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • Clofarabine — DRUG
    Clofarabine
  • Fludarabine — DRUG
    Fludarabine
  • Busulfan — DRUG
    Busulfan per PK
  • Cyclosporine-A — DRUG
    GVHD prophylaxis will consist of cyclosporine-A (CSA) and mycophenolate mofetil (MMF) starting day -3.
  • Mycophenolate Mofetil — DRUG
    GVHD prophylaxis will consist of cyclosporine-A (CSA) and mycophenolate mofetil (MMF) starting day -3.
  • Cord Blood Graft — BIOLOGICAL
    The CB graft will be infused on day 0 per standard practice

Study Details

This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.

Key Dates

Start date
Nov 20, 2020
Status verified
Mar 2026
Primary completion
Dec 20, 2026
Completion
Dec 20, 2026

Study Design

Enrollment
31 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Participants with malignant hematologic disorders
    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.
  • Experimental: Participants with non-malignant hematologic disorders
    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.

Primary Outcome Measure

Treatment related mortality at 1 year after myeloablative cord transplant [ Time Frame: 1 year ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Memorial Sloan Kettering Cancer CenterNew YorkNew York10065
Maria Cancio, MD
212-639-2446
E.

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