drugset / Trial / NCT00920972
Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases
Phase 1/2
Recruiting
220 enrolled
Washington University School of Medicine
St. Louis Children's Hospital · collab
Non-randomizedParallel-groupOpen-labelTreatment
Summary
The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient for engraftment of donor hematopoietic cells. It is also to determine major toxicities from these conditioning regimens, within the first 100 days after transplantation.
Timeline
- Start
- 2001-12
- Primary completion
- 2026-12
- Completion
- 2031-12
Publications
- Results Bhatla D, Davies SM, Shenoy S, Harris RE, Crockett M, Shoultz L, Smolarek T, Bleesing J, Hansen M, Jodele S, Jordan M, Filipovich AH, Mehta PA. Reduced-intensity conditioning is effective and safe for transplantation of patients with Shwachman-Diamond syndrome. Bone Marrow Transplant. 2008 Aug;42(3):159-65. doi: 10.1038/bmt.2008.151. Epub 2008 May 26.
- Results Hansen MD, Filipovich AH, Davies SM, Mehta P, Bleesing J, Jodele S, Hayashi R, Barnes Y, Shenoy S. Allogeneic hematopoietic cell transplantation (HCT) in Hurler's syndrome using a reduced intensity preparative regimen. Bone Marrow Transplant. 2008 Feb;41(4):349-53. doi: 10.1038/sj.bmt.1705926. Epub 2007 Nov 19.
- Results Rao A, Kamani N, Filipovich A, Lee SM, Davies SM, Dalal J, Shenoy S. Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning. Blood. 2007 Jan 1;109(1):383-5. doi: 10.1182/blood-2006-05-025072. Epub 2006 Sep 21.
- Results Shenoy S, Grossman WJ, DiPersio J, Yu LC, Wilson D, Barnes YJ, Mohanakumar T, Rao A, Hayashi RJ. A novel reduced-intensity stem cell transplant regimen for nonmalignant disorders. Bone Marrow Transplant. 2005 Feb;35(4):345-52. doi: 10.1038/sj.bmt.1704795.
- King AA, Kamani N, Bunin N, Sahdev I, Brochstein J, Hayashi RJ, Grimley M, Abraham A, Dioguardi J, Chan KW, Douglas D, Adams R, Andreansky M, Anderson E, Gilman A, Chaudhury S, Yu L, Dalal J, Hale G, Cuvelier G, Jain A, Krajewski J, Gillio A, Kasow KA, Delgado D, Hanson E, Murray L, Shenoy S. Successful matched sibling donor marrow transplantation following reduced intensity conditioning in children with hemoglobinopathies. Am J Hematol. 2015 Dec;90(12):1093-8. doi: 10.1002/ajh.24183. Epub 2015 Oct 6.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Abatacept | Other / unclassified | 10 mg/kg | Intravenous |
| Subject | Methotrexate | Small molecule | 7.5 mg/m2 | Intravenous |
| Subject | Thiotepa | Other / unclassified | 8 mg/kg | Intravenous |
| Background | Alemtuzumab | Monoclonal antibody | — | Intravenous |
| Background | Cyclosporine | Peptide | — | — |
| Background | Fludarabine | Small molecule | 30 mg/m2 | Intravenous |
| Background | Hydroxyurea | Other / unclassified | 30 mg/kg | Oral |
| Background | MMF | Unknown | — | — |
| Background | Melphalan | Small molecule | 140 mg/m2 | Intravenous |
| Background | Tacrolimus | Small molecule | — | — |