drugset / Trial / NCT03587272
Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease
Phase 2
Recruiting
100 enrolled
Robert Nickel
Alberta Children's Hospital · collabAnn & Robert H Lurie Children's Hospital of Chicago · collabChildren's Hospital at Montefiore · collabColumbia University · collabLevine Children's Hospital · collabNationwide Children's Hospital · collabThe Hospital for Sick Children · collab
NaSingle-groupOpen-labelTreatment
Summary
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).
Timeline
- Start
- 2018-04-17
- Primary completion
- 2030-11
- Completion
- 2030-11
Publications
- Nickel RS, Abraham A, Chiang KY, Ali M, Rangarajan HG, Chaudhury S, Kent M, Odinakachukwu M, Kukadiya D, Malloy J, Zhang A, Hardy SJ, Guilcher GMT. Nonmyeloablative HLA-identical sibling transplant for sickle cell disease in children: a multicenter prospective study. Blood Adv. 2026 Jun 23;10(12):4160-4170. doi: 10.1182/bloodadvances.2025019567.
- 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 |
|---|---|---|---|---|
| Subject | Alemtuzumab | Monoclonal antibody | 1 mg/kg | Intravenous |
| Subject | Sirolimus | Small molecule | 1 mg/m2 | — |