RecruitingPhase 2NCT06358638

Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody

Sickle Cell Disease Transplant Using a Nonmyeloablative Approach: Adding Daratumumab for Patients With Anti-donor Red Cell AntibodY

Sponsor

Children's National Research Institute

Enrollment

12 participants

Start Date

Apr 3, 2024

Study Type

INTERVENTIONAL

Conditions

Sickle Cell Disease

Summary

This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell aplasia with an acceptable safety profile in patients with anti-donor red blood cell antibodies, achieving an event-free survival similar to transplanted patients without such antibodies.

Eligibility

Min Age: 2 YearsMax Age: 25 Years

Plain Language Summary

Simplified for easier understanding

This Phase 2 multicenter study is testing whether adding daratumumab — a drug that targets antibody-producing cells — before a bone marrow transplant can prevent a rare complication called pure red blood cell aplasia in sickle cell disease patients who have antibodies against their donor's red blood cells. Without this intervention, these patients are often excluded from the least-toxic curative transplant approach available. Children and young adults aged 2 to 24 with sickle cell disease who have a matched sibling donor but have major ABO blood group incompatibility or other red blood cell alloantibodies may be eligible if they meet organ function and disease severity criteria. Participation involves receiving daratumumab before the transplant conditioning regimen, then proceeding with the bone marrow transplant and close follow-up monitoring. This summary was prepared to help patients understand the study in plain language.

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

DRUGDaratumumab

Daratumumab is a cytotoxic monoclonal antibody (IgG1k) to CD38 and is commercially approved to treat multiple myeloma. CD38 is expressed on several types of blood cells including B-cells, antibody-secreting plasma blasts and plasma cells. As a result, it has been used to treat antibody-mediated diseases, including children with antibody-mediated cytopenias post-HCT.

DRUGAlemtuzumab

Alemtuzumab is a humanized monoclonal antibody specific to lymphocyte antigens. It is a recombinant DNA-derived humanized monoclonal antibody (Campath-1H) that is directed against the 21-28 kD cell surface glycoprotein, CD52.

DRUGSirolimus

Sirolimus is an mTOR inhibitor immunosuppressant used to prevent organ transplant rejections as well as treat lymphangioleiomyomatosis and adults with perivascular epithelioid cell tumors.

RADIATIONTotal Body Irradiation

Total body irradiation is a form of radiotherapy used primarily as part of the preparative regimen for haematopoietic stem cell transplantation. The radiation is given in a low dose so that normal tissues can repair themselves.

Locations(1)

Children's National Hospital

Washington D.C., District of Columbia, United States

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NCT06358638

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