RecruitingPhase 1Phase 2NCT03173937

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

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

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Enrollment

37 participants

Start Date

Jun 13, 2017

Study Type

INTERVENTIONAL

Conditions

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

Summary

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.

Eligibility

Min Age: 4 YearsMax Age: 75 Years

Plain Language Summary

Simplified for easier understanding

This study tests a specialized cord blood transplant product called CordIn — which contains lab-grown stem cells from umbilical cord blood — in patients with severe aplastic anemia or related bone marrow failure conditions who have not responded to other treatments. The hope is that CordIn can help establish a healthy new blood and immune system while reducing the risk of graft failure that sometimes occurs with standard cord blood transplants. You may be eligible if: - You are between 4 and 60 years old - You have severe aplastic anemia with low bone marrow cellularity, or aplastic anemia that has evolved into a low-risk form of MDS - You have failed or cannot tolerate standard immunosuppressive therapy (like ATG and cyclosporine) - A suitable backup donor or backup cord blood unit is available in case the transplant does not work - A matched cord blood unit with sufficient cell counts has been identified You may NOT be eligible if: - A perfectly matched (12/12 HLA) related or unrelated donor is available - You are HIV positive - You have Fanconi anemia - You have significantly impaired lung, heart, liver, or kidney function - You have an active uncontrolled infection - You are pregnant or unwilling to use contraception - You have a history of malignant disease that could relapse within 5 years - You have antibodies against the donated cord blood cells (donor-specific antibodies) Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

BIOLOGICALOmidubicel (former CordIn)

Stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic umbilical/unrelated cord blood (UCB) cells. CordIn(TM) comprises: 1) cord blood-derived ex vivo expanded CD133+ cells (CordIn (TM) cultured fraction (CF)); and 2) the non-cultured cell fraction (CD133-) of the same CBU (CordIn(TM) Non-cultured Fraction (NF)). Both fractions, i.e. CordIn(TM) CF and CordIn(TM) NF are kept frozen until they are infused on the day of transplantation.

DEVICEMiltenyi CliniMACS(R) CD34 Reagent System

The CliniMACS CD34 Reagent System is a medical device system that is designed for the in vitro enrichment of CD34+ target cells from heterogeneous hematologic cell populations. The process of CD34+ cell selection results in simultaneous passive depletion of donor lymphocytes, potentially obviating the need for immunosuppressive drugs to prevent GVHD.