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Umbilical Cord Blood Transplant for Severe Aplastic Anemia and Hypo-plastic MDS

This study is testing a new approach to stem cell transplantation for people with severe aplastic anemia (SAA) or hypo-plastic myelodysplastic syndrome (MDS). Researchers are using a product called Omidubicel (formerly CordIn), which contains expanded stem cells from umbilical cord blood, along with a device called the Miltenyi CliniMACS CD34 Reagent System. The goal is to see if this combination can help patients recover faster after a transplant and improve their overall outcome. The study aims to enroll 37 participants between 4 and 75 years old. Success will be measured by how well the cord blood cells engraft (take hold) in the patient's body by day 100 after the transplant. The current status of the study is unclear.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It is designed to enroll 37 participants.
What's involved
You would undergo screening tests including blood, lung, and heart tests, a bone marrow biopsy, and a CT scan. You would also have an IV line placed and receive chemotherapy infusions and one radiation dose before the transplant.
Compensation
Not stated in the trial record.
Follow-up
The primary outcome, cord engraftment, is measured at or before day 100 after the transplant.

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NCT03173937

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

Recruiting
PHASE1Ages 4–75InterventionalTreatment
National Heart, Lung, and Blood Institute (NHLBI)
~37 participants
Updated 2026-08-26 on ClinicalTrials.gov
What's tested:Omidubicel (former CordIn)Miltenyi CliniMACS(R) CD34 Reagent System

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Cord engraftment
Measured over At or before day 100

Conditions

Severe Aplastic Anemia
Hypo-Plastic MDS
Myelodysplastic Syndrome (MDS)

Explore related trials

Myelodysplastic SyndromeSevere Aplastic Anemia

Where it's being run

1 sites across 1 states
Maryland1

Study leadership

  • Richard W Childs, M.D. · PRINCIPAL_INVESTIGATOR · National Heart, Lung, and Blood Institute (NHLBI)

Who to contact

Melissa M Spencer, R.N.
[email protected]
(301) 402-5609
Email the study team

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Eligibility criteria

Inclusion

Diagnosed with severe aplastic anemia with bone marrow cellularity \<30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and/or neutropenia (absolute neutrophil count \<=1000 cells/ uL or for patients receiving granulocyte transfusions, absolute neutrophil count \<=1000 cells/ uL before beginning granulocyte transfusions).
Intolerance of or failure to respond to immunosuppressive therapy. This also includes patients who have failed immunosuppressive therapy with ATG and cyclosporine or therapy with cyclosporine combined with eltrombopag in those who are intolerant of or do not have access to treatment with ATG.
Availability of at least one \>=4/8 HLA matched (HLA-A, B, C, and DR loci) cord blood unit from the National Marrow Donor Program (NMDP).
The CBU will have undergone volume reduction (both plasma and red blood cell depletion) prior to cryopreservation. All CBUs should be procured from public banks that meet local applicable regulations.
Ages 4-60 years inclusive.
Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects aged 4-17 years with formal consent being obtained from parents or legal guardian.

Exclusion

Availability of an HLA identical (12/12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft source and established donor selection factors (e.g. age, sex, viral exposure, ABO compatibility, pregnancy status, etc) per PI discretion.
ECOG performance status of 2 or more.
Major anticipated illness or organ failure incompatible with survival from transplant.
Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy, if of childbearing potential for one year.
HIV positive.
Diagnosis of Fanconi s anemia (by chromosome breakage study).
Diffusion capacity of carbon monoxide (DLCO) \<40% using DLCO corrected for Hgb or lung volumes (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO assessed).
Left ventricular ejection fraction \< 40% (evaluated by ECHO).
Transaminases \> 5x upper limit of normal.
Serum bilirubin \>4 mg/dl.
Creatinine clearance \< 50 cc/min/BSAm2 by 24-hour urine collection adjusted by body surface area.
Serum creatinine \> 2.5 mg/dl
Presence of an active infection not adequately responding to appropriate therapy.
History of a malignant disease liable to relapse or progress within 5 years.
Allergy to bovine, Gentamicin, or to any product which may interfere with the treatment.
Presence of donor-specific antibodies (DSA) to the umbilical cord blood unit and for cohort 1, to the haplo-identical donor.

What this trial measures

  • Cord engraftmentAt or before day 100

    Cord engraftment