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Stem Cell Transplantation

Umbilical Cord Blood Transplant for Aplastic Anemia and Myelodysplastic Syndrome

Phase 1 & 2

Recruiting

Led By Richard W Childs, M.D.

Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

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)

Must not have

History of a malignant disease liable to relapse or progress within 5 years

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 100 day and 200 day

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing 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.

Who is the study for?

This trial is for people aged 4-60 with severe aplastic anemia or myelodysplastic syndrome (MDS) who haven't responded to standard treatments. They need a matching cord blood unit and must understand the study's nature. Excluded are those with certain heart, kidney, liver conditions, active infections, other cancers within 5 years, specific allergies, HIV positive status or if they're pregnant.

What is being tested?

The trial tests a new treatment method using Omidubicel (formerly CordIn), which involves co-infusing stem cells from both a family member and unrelated donor cord blood to improve bone marrow transplant outcomes in patients with SAA or MDS.

What are the potential side effects?

Potential side effects may include reactions related to chemotherapy and radiation such as nausea, fatigue, infection risk increase due to immune suppression; complications from the stem cell infusion like graft-versus-host disease; and organ inflammation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My cord blood unit was processed and stored according to required standards.

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I have severe aplastic anemia, need regular blood or platelet transfusions, and have very low neutrophil counts.

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My severe aplastic anemia has turned into MDS with specific risk and bone marrow cellularity.

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I did not respond well to standard immune system treatments.

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My cord blood unit meets the required cell count criteria.

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I have a cord blood match of at least 4/8 HLA types from the NMDP.

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I am between the ages of 4 and 60.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a history of cancer that could come back or get worse within 5 years.

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I have a relative who is a complete or near-complete match for stem cell donation.

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I need some help with my daily activities.

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I do not have any major illnesses that would prevent me from surviving a transplant.

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My kidney function is reduced, with a creatinine clearance rate below 50.

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I have been diagnosed with Fanconi anemia.

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I have antibodies against the donor for my stem cell transplant.

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I have an infection that isn't getting better with treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 100 day and 200 day

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~100 day and 200 day

This trial's timeline: 3 weeks for screening, Varies for treatment, and 100 day and 200 day for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Cord engraftment

Secondary study objectives

Treatment related mortality (TRM), and standard transplant outcome variables such as non-hematologic toxicity, incidence and severity of acute and chronic GVHD, and relapse of disease