drugset / Trial / NCT02766465
Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)
Phase 2
Completed
138 enrolled
Medical College of Wisconsin
Blood and Marrow Transplant Clinical Trials Network · collabDana-Farber Cancer Institute · collabEmory University · collabNational Heart, Lung, and Blood Institute (NHLBI) · collabNational Marrow Donor Program · collab
Non-randomizedParallel-groupOpen-labelTreatment
Summary
This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care. The primary outcome is 2-year overall survival.
Timeline
- Start
- 2017-03-16
- Primary completion
- 2023-05-02
- Completion
- 2023-08-02
Publications
- Results Walters MC, Eapen M, Liu Y, El Rassi F, Waller EK, Levine JE, Strouse JJ, Antin JH, Parikh SH, Bakshi N, Dampier C, Jaroscak JJ, Bergmann S, Wong T, Kota V, Pace B, Lekakis LJ, Lulla P, Nickel RS, Kasow KA, Popat U, Smith W, Yu L, DiFronzo N, Geller N, Kamani N, Klings ES, Hassell K, Mendizabal A, Sullivan K, Neuberg D, Krishnamurti L. Hematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease. Blood Adv. 2025 Mar 11;9(5):955-965. doi: 10.1182/bloodadvances.2024013926.
- Leonard A, Furstenau D, Abraham A, Darbari DS, Nickel RS, Limerick E, Fitzhugh C, Hsieh M, Tisdale JF. Reduction in vaso-occlusive events following stem cell transplantation in patients with sickle cell disease. Blood Adv. 2023 Jan 24;7(2):227-234. doi: 10.1182/bloodadvances.2022008137.
- Krishnamurti L, Neuberg DS, Sullivan KM, Kamani NR, Abraham A, Campigotto F, Zhang W, Dahdoul T, De Castro L, Parikh S, Bakshi N, Haight A, Hassell KL, Loving R, Rosenthal J, Smith SL, Smith W, Spearman M, Stevenson K, Wu CJ, Wiedl C, Waller EK, Walters MC. Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study. Am J Hematol. 2019 Apr;94(4):446-454. doi: 10.1002/ajh.25401. Epub 2019 Feb 11.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Background | Alemtuzumab | Monoclonal antibody | 0.3 mg/kg | Intravenous |
| Background | Alemtuzumab | Monoclonal antibody | 20 mg | Intravenous |
| Background | Busulfan | Small molecule | 3.2 mg/kg | Intravenous |
| Background | Fludarabine | Small molecule | 30 mg/m2 | Intravenous |
| Background | Fludarabine | Small molecule | 35 mg/m2 | Intravenous |
| Background | G-CSF | Unknown | 5 ug/kg | — |
| Background | Melphalan | Small molecule | 140 mg/m2 | Intravenous |
| Background | Methotrexate | Small molecule | 7.5 mg/m2 | Intravenous |
| Background | Methotrexate | Small molecule | 10 mg/m2 | Intravenous |
| Background | Sirolimus | Small molecule | — | — |
| Background | Tacrolimus | Small molecule | — | — |
| Background | r-ATG | Unknown | 1.5 mg/kg | Intravenous |