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| Name | Class |
|---|---|
| Beth Israel Deaconess Medical Center | OTHER |
| Massachusetts General Hospital | OTHER |
| Brigham and Women's Hospital | OTHER |
| Emory University |
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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.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Busulfex | Drug | Given once daily for 4 days | ||
| Fludarabine | Drug | Given intravenously once daily for 4 days | ||
| Alemtuzumab | Drug | One day before fludarabine and busulfex are started, alemtuzumab will be given once daily for 5 days. |
| |
| Stem Cell Transfusion | Procedure | Performed three days after the end of chemotherapy |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Solid Organ Toxicity Related to the Conditioning Regimen. | Outcome was measured by the assessment of organ toxicity related to Busulfex, fludarabine and alemtuzumab. | 3 years |
| The Incidence of Grade II-IV Acute Graft vs. Host Disease. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Catherine J. Wu, MD | Dana-Farber Cancer Institute | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Winship Cancer Institute-Emory University | Atlanta | Georgia | 30322 | United States | ||
| Feist-Weiller Cancer Center-LSU |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 18550258 | Result | 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. |
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All enrolled patients received a stem cell transplant.
Activated for enrollment 3/4/2004. Closed to enrollment 4/25/2008. Participating institutions included: Dana-Farber Cancer Institute, Boston, Massachusetts, Feist-Weiller Cancer Center, LSU Health Sciences Center, Shreveport, Louisiana and Winship Cancer Institute, Emory University, Atlanta, Georgia
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| ID | Title | Description |
|---|---|---|
| FG000 | Transplant for Severe Hemoglobinopathies | Patients with severe hemoglobinopathies (eg. sickle cell disease, thalassemia major) with related donors who are identical at 6 HLA loci: (HLA-A, HLA-B, HLA-DRB1). The preparative regimen consisted of Busulfex, fludarabine and alemtuzumab. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| OTHER |
| Feist-Weiller Cancer Center at Louisiana State University Health Sciences | OTHER |
| Ohio State University | OTHER |
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Outcome was measured by incidence and severity of acute and chronic GVHD following donor stem cell infusion.
| 3 years |
| 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 |
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Transplant for Severe Hemoglobinopathies | Patients with severe hemoglobinopathies (eg. sickle cell disease, thalassemia major) with related donors who are identical at 6 HLA loci: (HLA-A, HLA-B, HLA-DRB1). The preparative regimen consisted of Busulfex, fludarabine and alemtuzumab. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||||
| Age Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | All patients enrolled. | Posted | Number | participants | 3 years |
|
|
| ||||||||||||||||||||||||||
| Secondary | Solid Organ Toxicity Related to the Conditioning Regimen. | Outcome was measured by the assessment of organ toxicity related to Busulfex, fludarabine and alemtuzumab. | All patients enrolled. | Posted | Number | participants | 3 years |
|
| |||||||||||||||||||||||||||
| Secondary | The Incidence of Grade II-IV Acute Graft vs. Host Disease. | Outcome was measured by incidence and severity of acute and chronic GVHD following donor stem cell infusion. | All patients enrolled. | Posted | Number | participants | 3 years |
|
|
Through study completion in 2009 (or patient death)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Transplant for Severe Hemoglobinopathies | Patients with severe hemoglobinopathies (eg. sickle cell disease, thalassemia major) with related donors who are identical at 6 HLA loci: (HLA-A, HLA-B, HLA-DRB1). The preparative regimen consisted of Busulfex, fludarabine and alemtuzumab. | 1 | 2 | 1 | 2 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Graft versus host disease | Immune system disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infection | Infections and infestations | Systematic Assessment |
|
Early termination leading to small numbers of subjects analyzed.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Catherine Wu, MD | Dana-Farber Cancer Institute | 617-632-5943 | cwu@partners.org |
| ID | Term |
|---|---|
| D006453 | Hemoglobinopathies |
| D000755 | Anemia, Sickle Cell |
| D013789 | Thalassemia |
| D006450 | Hemoglobin SC Disease |
| D006086 | Graft vs Host Disease |
| ID | Term |
|---|---|
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D002066 | Busulfan |
| C024352 | fludarabine |
| D000074323 | Alemtuzumab |
| ID | Term |
|---|---|
| D002072 | Butylene Glycols |
| D006018 | Glycols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
| D008698 | Mesylates |
| D000476 | Alkanesulfonates |
| D017738 | Alkanesulfonic Acids |
| D000473 | Alkanes |
| D006839 | Hydrocarbons, Acyclic |
| D006838 | Hydrocarbons |
| D013451 | Sulfonic Acids |
| D013456 | Sulfur Acids |
| D013457 | Sulfur Compounds |
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
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