Actively Recruiting
Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn, 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
252 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are serious bone marrow diseases often requiring bone marrow transplants. Researchers are studying a new method using a combination of blood stem cells from a family member and umbilical cord blood stem cells from an unrelated donor to see if this co-infusion is safe and effective for people aged 4 to 60 with these conditions. This research is motivated by the need to improve transplant outcomes and reduce graft rejection, which can occur in up to half of the cases with current treatments. Participants will receive a treatment involving CordIn(TM), a product consisting of purified and expanded umbilical cord blood stem and progenitor cells. Before transplantation, recipients undergo chemotherapy infusions and a single radiation dose starting 11 days prior. Donor cells are then infused through an IV line placed in the neck. Donors will be given injections to stimulate stem cell production and undergo apheresis to collect stem cells. Recipients stay in the hospital for 3 to 4 weeks post-transplant, followed by frequent outpatient visits for 3 to 4 months, then visits every 6 months for five years. During the study, recipients will have various tests including blood, lung, heart evaluations, bone marrow biopsy, and CT scans. Donors are screened with physical exams, medical history review, and blood tests. Researchers will monitor engraftment success by day 100 as the primary outcome and assess treatment-related mortality, transplant complications, graft-versus-host disease, disease relapse, and quality of life as secondary outcomes. The total study participation includes long-term follow-up for up to five years after transplant discharge.
CONDITIONS
Brief 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 transfusion dependence and/or neutropenia
- History of severe aplastic anemia transformed to MDS meeting specific IPSS risk and marrow criteria
- Intolerance of or failure to respond to immunosuppressive therapy including ATG and cyclosporine
- Availability of at least one alternative donor (haploidentical or matched unrelated donor) for salvage transplant
- Availability of at least one cord blood unit matched at least 4/8 HLA loci with specified cell doses
- Cord blood units must meet minimum cell counts or have approval for expansion use
- Age between 4 and 60 years inclusive
- Ability to understand the study and provide informed consent (parental consent for ages 4-17)
You will not qualify if you...
- Availability of an HLA identical (12/12) matched related or unrelated donor within 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
- HIV positive status
- Diagnosis of Fanconi anemia
- Lung diffusion capacity (DLCO) less than 40% or inability to perform test if under 10 years
- Left ventricular ejection fraction less than 40%
- Elevated liver enzymes or bilirubin beyond specified limits
- Reduced kidney function below specified thresholds
- Presence of active infection not responding to therapy
- History of malignancy likely to relapse or progress within 5 years
- Allergy to bovine, gentamicin, or interfering products
- Presence of donor-specific antibodies to cord blood unit or haploidentical donor for cohort 1
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 100 days or until engraftment is confirmed
Participants undergo transplantation with CordIn(TM), an ex vivo expanded umbilical cord blood stem/progenitor cell product, to expedite engraftment and improve transplant outcome for severe aplastic anemia or hypoplastic MDS.
Multiple visits during transplantation and recovery period
Duration - Up to 200 days post-transplant
Participants are monitored for treatment-related mortality, graft-versus-host disease, relapse, and other transplant outcomes up to 200 days post-transplant.
Regular visits for assessments up to 200 days
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.
R
Richard W Childs, M.D.
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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