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Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease

Study on new treatment options for sickle cell disease.

Recruiting
6-21 years
All
Phase 2
Study Overview: This research is a follow-up trial to evaluate how safe and effective it is to use a medicine called Defibrotide for patients with sickle cell disease or beta thalassemia who are getting a special type of stem cell transplant. These patients are at risk for a liver problem called sinusoidal obstructive syndrome (SOS), and Defibrotide might help prevent it. Key Terms: - **Myeloablative Conditioning**: A strong treatment to prepare the body for a stem cell transplant. - **Haplo AlloSCT**: A stem cell transplant using a family member who is a half-match. - **Defibrotide Prophylaxis**: Using Defibrotide medicine to prevent a disease. - **CD34 Enrichment**: A way to make the stem cells used in the transplant more effective. - **T-cell Addback**: Adding T-cells back into the body to help fight infections. Key Points:
  • This study involves a stem cell transplant and uses Defibrotide to help prevent liver problems.
  • Participants must have specific complications from sickle cell disease or beta thalassemia to join.
  • Women who are pregnant or breastfeeding cannot take part in this study.
Study details
    Sickle Cell Disease

NCT02675959

New York Medical College

22 August 2026

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