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Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

Stem cell transplant trial for sickle cell patients with low donor cells.

Recruiting
2-80 years
All
Phase 1/2
Study Overview: This study is for people with sickle cell disease (a blood disorder) who already had a blood stem cell transplant but experienced a return of symptoms. The goal is to give a second transplant to help cure the disease by making more space in the bone marrow for healthy donor cells. Key Points:
  • The study involves a stay in the hospital for about 30 days after receiving the donor cells.
  • Participants will have frequent visits in the first 3 months, and then every 6 months to 1 year for 5 years.
  • Donors must visit the clinic 7-8 times and take a medicine for 5-6 days before donation.
Eligibility: Participants must be at least 4 years old and already had a transplant. Donors, who are relatives and can be as young as 2, will donate blood stem cells. Recipients will undergo various tests, and donors will prepare with medication before donating. Risks & Considerations: Potential risks include infection or rejection of the transplant. It's important for recipients to have a caregiver and be ready for a lengthy observation period.
Study details
    Myeloid Chimerism

NCT04008368

National Heart, Lung, and Blood Institute (NHLBI)

17 April 2026

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