Actively Recruiting
Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies and Non-malignant Disorders
Led by Memorial Sloan Kettering Cancer Center · Updated on 2026-03-13
31
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying children and young adults with life-threatening blood cancers and non-malignant blood disorders who do not have a matched related donor for allogeneic transplantation. This phase 2 trial aims to investigate the treatment-related mortality one year after receiving a myeloablative cord blood transplant. The study focuses on both malignant and non-malignant hematologic conditions to better understand outcomes in this vulnerable population. Participants with malignant disorders receive chemotherapy drugs including clofarabine, fludarabine phosphate, and busulfan before the transplant. They also receive immune suppression drugs tacrolimus and mycophenolate mofetil starting several days before the transplant to help prevent graft versus host disease (GVHD). Patients with non-malignant disorders receive additional treatments such as rituximab and rabbit anti-thymocyte globulin before chemotherapy and transplant. All patients undergo cord blood transplantation on day 0, followed by supportive care including filgrastim injections to aid blood cell recovery and additional rituximab for non-malignant cases. Throughout the study, participants have regular blood tests, and imaging scans including computed tomography (CT) and positron emission tomography (PET) to monitor their condition. Researchers track treatment-related mortality and recovery over one year after the transplant. The study involves careful monitoring of organ function, immune response, and overall health status. Participants may have medication adjustments based on their response, with the goal of understanding safety and outcomes in this treatment approach.
CONDITIONS
Brief Title
Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 21 years or younger at time of consent
- No available and suitably matched related or unrelated donor
- Diagnosed with specific hematologic malignancies such as high-risk AML or ALL, other acute leukemias with low blast counts, MDS/MPD without myelofibrosis, NHL or HL at high risk, inherited metabolic disorders, or non-malignant disorders including hemoglobinopathies, bone marrow failure, and immunodeficiencies
- Karnofsky or Lansky score of at least 70%
- Bilirubin no greater than 1.5 mg/dL (unless benign congenital hyperbilirubinemia)
- ALT no greater than three times upper limit of normal
- Pulmonary function at or above 50% predicted
- Left ventricular ejection fraction at or above 50%
- Age-adjusted Hematopoietic Cell Transplantation-Comorbidity Index less than or equal to 7
- Renal function within specified normal ranges
- Cord blood units meeting HLA matching and cell dose criteria as per selection algorithm
You will not qualify if you...
- Inadequate performance status or organ function
- Advanced metabolic disease
- Active central nervous system leukemic involvement
- Indolent NHL or HL with progressing disease after recent chemotherapy
- Diagnosis of myelofibrosis or malignancy with moderate to severe bone marrow fibrosis
- Autologous stem cell transplant within past 6 months
- Any prior allogeneic stem cell transplant
- Active, uncontrolled infections at time of transplant
- HIV infection
- Seropositivity for HTLV-1
- Pregnancy or breastfeeding
- Inability to give informed consent or comply with study protocol including follow-up and research tests
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - From approximately day -12 to day 30 post-transplant
Participants receive conditioning chemotherapy including clofarabine, fludarabine, and busulfan, followed by cord blood transplantation on day 0. They begin immunosuppressive therapy with tacrolimus and mycophenolate mofetil starting day -3 to prevent graft versus host disease (GVHD). Patients with non-malignant disorders also receive rituximab and rabbit anti-thymocyte globulin prior to conditioning. Post-transplant, participants receive filgrastim starting day 7 until neutrophil recovery.
Multiple visits including infusion and monitoring visits during conditioning and transplantation
Duration - Up to 1 year after transplant
Participants are monitored for treatment-related mortality and graft versus host disease. Immunosuppressive medications may be tapered approximately 2 to 6 months post-transplant in the absence of ongoing GVHD. Blood samples and imaging studies such as CT and PET scans are collected for ongoing assessment.
Regular follow-up visits including blood sample collection and imaging assessments
Trial Site Locations
Total: 1 location
1
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
Actively Recruiting
Research Team
M
Maria I Cancio, MD
J
Jaap Jan Boelens, MD, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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