RecruitingPhase 1Phase 2NCT03653338

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent Anemias


Sponsor

Paul Szabolcs

Enrollment

5 participants

Start Date

Aug 2, 2018

Study Type

INTERVENTIONAL

Conditions

Summary

The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.


Eligibility

Min Age: 5 YearsMax Age: 40 Years

Plain Language Summary

Simplified for easier understanding

This study offers bone marrow transplantation using donors who are not a perfect tissue match (alternative donors) for patients with sickle cell disease or other serious anemias such as beta-thalassemia and Diamond-Blackfan anemia. Standard bone marrow transplants require a closely matched sibling donor, which many patients don't have. This trial uses a special technique called T-cell depletion to reduce the risk of rejection and graft-versus-host disease, making transplants from partially matched donors safer. The goal is to give patients without a perfect donor a chance at a cure. The transplant can eliminate or greatly reduce the need for blood transfusions and prevent serious complications like strokes, pain crises, and organ damage caused by sickle cell disease. You may be eligible if: - You are between 5 and 40 years of age - You have sickle cell disease (HbSS or Sβ0-thalassemia) with serious complications such as recurrent pain crises, acute chest syndrome, stroke, or organ damage - OR you have beta-thalassemia or Diamond-Blackfan anemia requiring regular blood transfusions - You do not have a fully matched sibling or unrelated donor available - Your organs (heart, lungs, liver, kidneys) are functioning adequately - You have tried and failed hydroxyurea (for sickle cell disease) You may NOT be eligible if: - You have a fully matched sibling or matched unrelated donor available - You have had a stem cell transplant within the past 6 months - You have had a central nervous system event (e.g., stroke) within the past 6 months - You are pregnant or breastfeeding - You have an active, uncontrolled infection 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

BIOLOGICALCD3/CD19 depleted leukocytes

Negative selection for CD3+/CD19+ cells will be performed on the CliniMACS® depletion device.

BIOLOGICALCD45RA depleted leukocytes

Negative selection for CD45RA will be performed on the CliniMACS® depletion device.

DRUGHydroxyurea

Sickle Cell Disease Conditioning

DRUGRituximab

Sickle Cell Disease Conditioning

DRUGAlemtuzumab

Sickle Cell Disease Conditioning

DRUGFludarabine

Sickle Cell Disease Conditioning

DRUGThiotepa

Sickle Cell Disease Conditioning


Locations(1)

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

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NCT03653338


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