This trial is testing a new way to prepare for a bone marrow transplant that may be less intense and have fewer side effects.
- Non-Malignant Disorders
- Hemoglobin Disorders
- Metabolic Disorders
- Blood Disorders
2 Primary · 6 Secondary · Reporting Duration: 2 years post-transplant
4 Treatment Groups
1 of 4
1 of 4
1 of 4
1 of 4
220 Total Participants · 4 Treatment Groups
Primary Treatment: GVHD Regimen B: BM Recipients · No Placebo Group · Phase 1 & 2
Who is running the clinical trial?
Age < 65 · All Participants · 5 Total Inclusion CriteriaMark “Yes” if the following statements are true for you:
- Bhatla, D, S M Davies, S Shenoy, R E Harris, M Crockett, L Shoultz, T Smolarek, et al.. 2008. “Reduced-intensity Conditioning Is Effective and Safe for Transplantation of Patients with Shwachman–diamond Syndrome”. Bone Marrow Transplantation. Springer Science and Business Media LLC. doi:10.1038/bmt.2008.151.
- Hansen, M D, A H Filipovich, S M Davies, P Mehta, J Bleesing, S Jodele, R Hayashi, Y Barnes, and S Shenoy. 2007. “Allogeneic Hematopoietic Cell Transplantation (HCT) in Hurler's Syndrome Using a Reduced Intensity Preparative Regimen”. Bone Marrow Transplantation. Springer Science and Business Media LLC. doi:10.1038/sj.bmt.1705926.
- Shenoy, S, W J Grossman, J DiPersio, L C Yu, D Wilson, Y J Barnes, T Mohanakumar, A Rao, and R J Hayashi. 2004. “A Novel Reduced-intensity Stem Cell Transplant Regimen for Nonmalignant Disorders”. Bone Marrow Transplantation. Springer Science and Business Media LLC. doi:10.1038/sj.bmt.1704795.
- Rao, Aarati, Naynesh Kamani, Alexandra Filipovich, Susan Molleran Lee, Stella M. Davies, Jignesh Dalal, and Shalini Shenoy. 2006. “Successful Bone Marrow Transplantation for IPEX Syndrome After Reduced-intensity Conditioning”. Blood. American Society of Hematology. doi:10.1182/blood-2006-05-025072.
- King, Allison A., Naynesh Kamani, Nancy Bunin, Indira Sahdev, Joel Brochstein, Robert J. Hayashi, Michael Grimley, et al.. 2015. “Successful Matched Sibling Donor Marrow Transplantation Following Reduced Intensity Conditioning in Children with Hemoglobinopathies”. American Journal of Hematology. Wiley. doi:10.1002/ajh.24183.
- 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.
- 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.
- 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.
- 2001. "Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT00920972.
Frequently Asked Questions
Is this study still open to new participants?
"That is correct, the online information indicates that this research is looking for more subjects. This trial was first advertised on December 1st 2001 and updated October 12th 2021. The aim is to have 220 people enrolled at 20 different hospitals or clinics." - Anonymous Online Contributor
What is the projected class size for this trial?
"That is accurate. The latest information on clinicaltrials.gov reveals that this trial, which was first announced on December 1st 2001, is still looking for participants. They need to enroll 220 patients from 20 different locations." - Anonymous Online Contributor
What is the purpose of this clinical trial?
"The purpose of this 100-day clinical trial is to evaluate the major toxicities of a new transplant treatment. Secondary outcomes include immune reconstitution, T cell function, B cell function, and incidence of acute graft-versus-host disease. Finally, the time to neutrophil and platelet engraftment will be measured." - Anonymous Online Contributor
Is this trial being held in more than one state?
"In addition to All Children's Hospital in Saint Petersburg, Florida, Louisiana State University in New Orleans, Louisiana, and St. Louis University in Saint Louis, Oklahoma, this clinical trial is also enrolling patients at 20 other locations." - Anonymous Online Contributor