A Blood Stem Cell Transplant for Sickle Cell Disease (NCT03249831)

🧪 Interventions in This Study

• Cyclophosphamide (DRUG)
• Pentostatin (DRUG)
• Rabbit anti-thymocyte globulin (DRUG)
• Tacrolimus (DRUG)
• Mycophenolate mofetil (DRUG)
• CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant (BIOLOGICAL)

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

1. Confirmed diagnosis of hemoglobin SS or S-βº Thalassemia sickle cell disease
2. Severe disease status as defined by presence of one or more of the following:
3. Clinically significant neurologic event (stroke) or any neurological deficit lasting \> 24 hours; or increased transcranial Doppler velocity (\>200 m/s). A stroke is defined as a sudden neurologic change lasting more than 24 hours that is accompanied by cerebral magnetic resonance imaging (MRI) changes.
4. History of ≥ 1 episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea).
5. History of ≥ 2 severe vaso-occlusive pain crises (VOC) per year in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. a pain management plan and/or treatment with hydroxyurea). A severe VOC is defined as an episode of pain lasting more than 2 hours severe enough to require care at a medical facility. Note that priapism that lasts more than 2 hours and requires care at a medical facility is also considered a VOC.
6. Osteonecrosis of ≥ 2 joints despite the institution of supportive care measures.
7. Prior treatment with regular RBC transfusion therapy, defined as receiving ≥ 8 transfusions per year for \> 1 year to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and acute chest syndrome)
8. No HLA matched sibling or 10/10 matched unrelated donor
9. Related donor who:
10. Is genotypically haploidentical on HLA-A, B, C and DRB1 loci AND
11. Meets institutional criteria
12. Failed prior hydroxyurea therapy or have intolerance to hydroxyurea

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (1 Sites)

• City of Hope Medical Center — Duarte, California

📞 Contact the Study Coordinator

Phone: (800) 826-4673

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

💰 Cost, Insurance & Patient Rights

• 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
• Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
• Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

More studies in Sickle Cell Disease

• Effect of Virtual Reality Technology for Pain Management of Vaso-Occlusive Crisis in Patients With Sickle Cell Disease — Memphis, Tennessee
• Kidney Function in Sickle Cell Anemia — Chapel Hill, North Carolina
• BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease — Birmingham, Alabama
• Virtual Reality for SCD VOC — Baltimore, Maryland
• Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease — Bethesda, Maryland
• Acupressure in Patients With Sickle Cell Disease — Irvine, California

Explore by condition: All Sickle Cell Disease clinical trials

💡 How to Participate

1. Check the checklist above to see if you may qualify.
2. Contact the study coordinator using the phone/email above.
3. Ask about the visit schedule, what is covered at no cost, and any travel support.
4. Confirm with your own doctor before making a decision.