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

What's the purpose of this trial?

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 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.

This trial is currently open and accepting patients.

What will happen during the trial?

You may be able to join this trial if you:

The following criteria is a partial list of reasons why patients may be eligible to participate in this clinical trial. Further evaluation with a medical professional is required.

* 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).


History of severe aplastic anemia transformed to MDS that meet the following criteria: a) International Prognostic Scoring System (IPSS) risk category of INT-1 or greater, b) \< 5% myeloblasts and \< 30% of cellularity in the bone marrow on screening morphologic analysis.

* Intolerance of or failure to respond to standard immunosuppressive therapy.
* Identification of either a) at least one alternative donor (i.e. HLA- haploidentical related donor (i.e. greater than or equal to 5/10 HLA match: HLA-A, B, C, DR, and DQ loci) or greater than or equal to 9/10 HLA matched unrelated donor) who is available to serve as a stem cell donor for a salvage allogeneic transplant in the event that the CordIn unit has been rejected or b) umbilical cord blood unit/s that can be used for a salvage cord blood transplant in the event that the CordIn unit has been rejected.
* Availability of at least one greater than or equal to 4/8 HLA-matched (HLA-A, B, C, and DR loci) cord blood unit from the National Marrow Donor Program (NMDP).
* The cord blood unit must contain a minimum TNC of at least 1.8 x 10\^9 and at least 1.5x10\^7/kg TNC and at least 8 x 10\^6 CD34+ cells (all doses prior to thawing.

Exception: Cord units containing at least 1.5x10\^7/kg TNC and at least 8 x 10\^6 CD34+ cells but less than 1.8 x 10\^9 TNC may be eligible for use on this trial if the total TNC is a) at least 1.5 x 10\^9 AND b) approval for use of this cord unit for expansion is granted by Gamida Cell.

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.


* Availability of an HLA identical or 9/10 HLA matched (HLA A, B, C, DR, and DQ loci) - relative to serve as a stem cell donor.
* The patient is deemed to be a candidate for a 10/10 HLA matched unrelated stem cell transplant (availability of a donor and resources required for such a transplant).
* 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% predicted 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).

1-Transaminases \> 5x upper limit of normal.
* Serum bilirubin \>4 mg/dl.
* Creatinine clearance \< 50 cc/min/BSAm\^2 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.

Additional Trial Information

Phase 1/2

Enrollment: 37 patients (estimated)

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National Institutes of Health (Bethesda)

Bethesda, MD

Open and Accepting
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