Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies and Non-malignant Disorders
1 other identifier
interventional
31
1 country
1
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Nov 2020
Longer than P75 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 20, 2020
CompletedFirst Submitted
Initial submission to the registry
November 23, 2020
CompletedFirst Posted
Study publicly available on registry
November 25, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 20, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 20, 2026
March 13, 2026
March 1, 2026
6.1 years
November 23, 2020
March 12, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Treatment related mortality at 1 year after myeloablative cord transplant
The primary objective of this study is to assess treatment related mortality (TRM) at 1 year after myeloablative cord transplant.
1 year
Study Arms (2)
Participants with malignant hematologic disorders
EXPERIMENTALPatients 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.
Participants with non-malignant hematologic disorders
EXPERIMENTALPatients 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.
Interventions
Fludarabine
Busulfan per PK
GVHD prophylaxis will consist of cyclosporine-A (CSA) and mycophenolate mofetil (MMF) starting day -3.
GVHD prophylaxis will consist of cyclosporine-A (CSA) and mycophenolate mofetil (MMF) starting day -3.
The CB graft will be infused on day 0 per standard practice
Clofarabine
Eligibility Criteria
You may qualify if:
- Age and Donor Status:
- Patients with age ≤ 21 years at time of consent with no available and suitably matched related or unrelated donor in the required time period.
- Diagnoses :
- I. Acute myelogenous leukemia (AML) :
- Complete first remission (CR1) at high risk for relapse such as any of the following:
- Known prior diagnosis of myelodysplasia (MDS) or myeloproliferative disorder (MPS).
- Therapy-related AML (t-AML).
- White cell count at presentation \> 100,000.
- Presence of extramedullary leukemia at diagnosis.
- Any unfavorable subtype by FAB or WHO classification.
- High-risk cytogenetics (e.g. those associated with MDS, abnormalities of 5, 7, 8, complex karyotype) or high-risk molecular abnormalities.
- Requirement for 2 or more inductions to achieve CR1.
- Presence of Minimal Residual Disease (MRD+) by cytogenetics, flow cytometry or molecular methods after induction.
- Any patient with newly diagnosed AML with intermediate risk cytogenetics who elects allograft with curative intent over consolidation chemotherapy.
- Any patient unable to tolerate consolidation chemotherapy as would have been deemed appropriate by the treating physician.
- +55 more criteria
You may not qualify if:
- Inadequate performance status/ organ function.
- Advanced metabolic disease (EBMT handbook).
- Active CNS leukemic involvement.
- Indolent NHL or Hodgkin lymphoma with progression of disease after most recent salvage chemotherapy.
- Diagnosis of myelofibrosis or other malignancy with moderate-severe bone marrow fibrosis.
- Autologous stem cell transplant within the preceding 6 months.
- Any prior allogeneic stem cell transplant.
- Active and uncontrolled infection (bacterial/fungal/viral) at time of transplantation.
- HIV infection.
- Seropositivity for HTLV-1.
- Pregnancy or breast feeding.
- Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, long-term follow-up, and research tests.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Maria Cancio, MD
Memorial Sloan Kettering Cancer Center
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 23, 2020
First Posted
November 25, 2020
Study Start
November 20, 2020
Primary Completion (Estimated)
December 20, 2026
Study Completion (Estimated)
December 20, 2026
Last Updated
March 13, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will share
Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.