Actively Recruiting
Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome
Led by National Heart, Lung, and Blood Institute (NHLBI) · Updated on 2026-04-06
37
Participants Needed
1
Research Sites
767 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant. Researchers are testing ways to make stem cell transplant safer and more effective. Objective: To test if treating people with SAA or MDS with a co-infusion of blood stem cells from a family member and cord blood stem cells from an unrelated donor is safe and effective. Eligibility: Recipients ages 4-60 with SAA or MDS Donors ages 4-75 Design: Recipients will be screened with: * Blood, lung, and heart tests * Bone marrow biopsy * CT scan Recipients will have an IV line placed into a vein in the neck. Starting 11 days before the transplant they will have several chemotherapy infusions and 1 30-minute radiation dose. Recipients will get the donor cells through the IV line. They will stay in the hospital 3-4 weeks. After discharge, they will have visits: * First 3-4 months: 1-2 times weekly * Then every 6 months for 5 years Donors will be screened with: * Physical exam * Medical history * Blood tests Donors veins will be checked for suitability for stem cell collection. They may need an IV line to be placed in a thigh vein. Donors will get Filgrastim or biosimilar (G-CSF) injections daily for 5-7 days. On the last day, they will have apheresis: Blood drawn from one arm or leg runs through a machine and into the other arm or leg. This may be repeated 2 days or 2-4 weeks later.
CONDITIONS
Official Title
Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with severe aplastic anemia with bone marrow cellularity less than 30% and requiring transfusions or with neutropenia
- Or history of severe aplastic anemia transformed to MDS with IPSS risk of INT-1 or greater, less than 5% myeloblasts, and less than 30% marrow cellularity
- Intolerance or failure to respond to immunosuppressive therapy including ATG and cyclosporine
- Availability of at least one alternative donor (haploidentical related donor with 5/10 or more HLA match or 9/10 or more HLA matched unrelated donor) for salvage transplant
- Availability of at least one 4/8 or greater HLA matched cord blood unit with minimum TNC and CD34+ cell doses
- Cord blood units must meet volume reduction and regulatory standards
- Ages 4 to 60 years inclusive
- Ability to understand investigational nature and provide informed consent (parents/guardians consent for ages 4-17)
You will not qualify if you...
- Availability of an HLA identical (12/12) matched related or unrelated donor available in optimal timeline
- ECOG performance status of 2 or higher
- Major illness or organ failure incompatible with transplant survival
- Current pregnancy or unwillingness to use birth control if of childbearing potential for one year
- HIV positive status
- Diagnosis of Fanconi anemia
- Diffusion capacity of carbon monoxide (DLCO) less than 40% (with exceptions for children under 10)
- Left ventricular ejection fraction less than 40%
- Liver transaminases more than 5 times upper limit of normal
- Serum bilirubin greater than 4 mg/dl
- Creatinine clearance less than 50 cc/min/BSAm2 or serum creatinine greater than 2.5 mg/dl
- Presence of active infection not responding to treatment
- History of malignant disease likely to relapse or progress within 5 years
- Allergy to bovine, gentamicin, or any product interfering with treatment
- Presence of donor-specific antibodies to the cord blood unit or haplo-identical donor for cohort 1
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
M
Melissa M Spencer, R.N.
CONTACT
R
Richard W Childs, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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