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 clinical trial is studying a drug called Alemtuzumab, a biological treatment called CD3/CD19 depleted leukocytes, and others for people with diamond-blackfan anemia, sickle cell anemia, and other related conditions. The study is currently recruiting participants at 1 location. People eligible for this study include aged 5 Years to 40 Years.

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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