drugset / Trial / NCT03263559
Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)
Phase 2
Completed
95 enrolled
Medical College of Wisconsin
Blood and Marrow Transplant Clinical Trials Network · collabNational Cancer Institute (NCI) · collabNational Heart, Lung, and Blood Institute (NHLBI) · collabNational Marrow Donor Program · collab
NaSingle-groupOpen-labelTreatment
Summary
This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1) children with severe SCD; and (2) adults with severe SCD.
Timeline
- Start
- 2017-10-03
- Primary completion
- 2025-01-29
- Completion
- 2025-01-29
Publications
- Results Hong S, Horwitz ME. Cures for Sickle Cell Abound - How about Access? NEJM Evid. 2025 Mar;4(3):EVIDe2400428. doi: 10.1056/EVIDe2400428. Epub 2025 Feb 25. No abstract available.
- Results Kassim AA, Walters MC, Eapen M, Smith M, Logan BR, Solh M, McKinney C, Nieder M, Ross M, Kent M, Abusin GA, Mallhi K, Silva JG, Shaughnessy P, Kanter J, Haines H, Farah R, Khaled YA, Ritzau N, Mendizabal A, Abraham A, Bollard C, Cooke K, de la Fuente J, Hanna R, Horowitz MM, Jordan LC, Bakshi N, Krishnamurti L, Leifer E, Mahadeo KM, Shenoy S, Jones RJ, DeBaun MR, Brodsky RA. Haploidentical Bone Marrow Transplantation for Sickle Cell Disease. NEJM Evid. 2025 Mar;4(3):EVIDoa2400192. doi: 10.1056/EVIDoa2400192. Epub 2025 Feb 25.
- 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.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Background | 2-MERCAPTOETHANESULFONIC ACID | Small molecule | 40 mg/kg | — |
| Background | Cyclophosphamide | Other / unclassified | 50 mg/kg | — |
| Background | Fludarabine | Small molecule | 30 mg/m2 | — |
| Background | Hydroxyurea | Other / unclassified | 30 mg/kg | — |
| Background | Thiotepa | Other / unclassified | 10 mg/kg | — |
| Background | Thymoglobulin | Protein / enzyme biologic | 2 mg/kg | — |