Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies
Multi-Center Study Using Allogeneic Stem Cell Transplantation Following Reduced Intensity Chemotherapy in Patients With Hemoglobinopathies
1 other identifier
interventional
2
1 country
6
Brief Summary
The purpose of this study is to determine if treatment with reduced-dose busulfex, fludarabine and alemtuzumab (CAMPATH) followed by sten cell infusion will allow for donor stem cells to grow in patients with hemoglobinopathies bone marrow and restore circulating blood counts. In addition the incidence and severity of side effects and of graft vs. host disease (GVHD) will be monitored.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Mar 2004
Longer than P75 for phase_2
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
March 1, 2004
CompletedFirst Submitted
Initial submission to the registry
September 8, 2005
CompletedFirst Posted
Study publicly available on registry
September 12, 2005
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2008
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2009
CompletedResults Posted
Study results publicly available
March 12, 2013
CompletedJuly 30, 2013
July 1, 2013
4 years
September 8, 2005
December 5, 2012
July 24, 2013
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy.
Outcome was measured by ANC \>500 for three consecutive days prior to day 30 after PBSC infusion, \>25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods prior to day 45 after PBSC infusion and \>25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods after day 180 after PBSC infusion.
3 years
Secondary Outcomes (2)
Solid Organ Toxicity Related to the Conditioning Regimen.
3 years
The Incidence of Grade II-IV Acute Graft vs. Host Disease.
3 years
Interventions
Given once daily for 4 days
Given intravenously once daily for 4 days
One day before fludarabine and busulfex are started, alemtuzumab will be given once daily for 5 days.
Performed three days after the end of chemotherapy
Eligibility Criteria
You may qualify if:
- Patients with sickle cell disease should have one or more of the following: acute chest syndrome requiring hospitalization; nonhemorrhagic stroke or central nervous system event lasting longer than 24 hours; recurrent caso-occlusive pain or recurrent priapism; sickle neuropathy; bilateral proliferative retinopathy and major visual impairment of at least one eye; osteonecrosis of multiple joints; transfusion dependence; vaso-occlusive.
- Patients with thalassemia should have one or more of the following: transfusion dependence; iron overload; presence of 2 or more alloantibodies against red cell antigens.
You may not qualify if:
- Pregnancy
- Acute hepatitis
- Cardiac ejection fraction \< 30%
- Severe renal impairment
- Severe residual functional neurologic impairment
- Evidence of HIV infection
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Dana-Farber Cancer Institutelead
- Beth Israel Deaconess Medical Centercollaborator
- Massachusetts General Hospitalcollaborator
- Brigham and Women's Hospitalcollaborator
- Emory Universitycollaborator
- Feist-Weiller Cancer Center at Louisiana State University Health Sciencescollaborator
- Ohio State Universitycollaborator
Study Sites (6)
Winship Cancer Institute-Emory University
Atlanta, Georgia, 30322, United States
Feist-Weiller Cancer Center-LSU
Shreveport, Louisiana, 71130, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02115, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115, United States
Massachusetts General Hospital
Boston, Massachusetts, 02115, United States
Ohio State University College of Medicine
Columbus, Ohio, 43210, United States
Related Publications (1)
Armistead PM, Mohseni M, Gerwin R, Walsh EC, Iravani M, Chahardouli B, Rostami S, Zhang W, Neuberg D, Rioux J, Ghavamzadeh A, Ritz J, Antin JH, Wu CJ. Erythroid-lineage-specific engraftment in patients with severe hemoglobinopathy following allogeneic hematopoietic stem cell transplantation. Exp Hematol. 2008 Sep;36(9):1205-15. doi: 10.1016/j.exphem.2008.04.004. Epub 2008 Jun 11.
PMID: 18550258RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
Early termination leading to small numbers of subjects analyzed.
Results Point of Contact
- Title
- Catherine Wu, MD
- Organization
- Dana-Farber Cancer Institute
Study Officials
- PRINCIPAL INVESTIGATOR
Catherine J. Wu, MD
Dana-Farber Cancer Institute
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Principal Investigator
Study Record Dates
First Submitted
September 8, 2005
First Posted
September 12, 2005
Study Start
March 1, 2004
Primary Completion
March 1, 2008
Study Completion
July 1, 2009
Last Updated
July 30, 2013
Results First Posted
March 12, 2013
Record last verified: 2013-07