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| Name | Class |
|---|---|
| Carelon Research | OTHER |
| Pediatric Blood and Marrow Transplant Consortium | OTHER |
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This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks and side effects.
This study includes a preparative regimen with Hydroxyurea, Alemtuzumab, Fludarabine, Thiotepa and Melphalan that provides intense host immunosuppression without myeloablation. The primary hypothesis is that this regimen will promote stable engraftment of unrelated donor hematopoietic cells, support normal erythropoiesis, and result in an event free survival of > 75% of children with thalassemia major.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Conditioning regimen | Experimental | Hydroxyurea days -50 to -21 Alemtuzumab days -21 to -19 Fludarabine days -8 to -4 Thiotepa day -4 Melphalan day -3 Stem cell infusion day 0 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan | Drug | Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Primary Objective: Event-free Survival at 1 Year. | 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Development of Graft Versus Host Disease (GVHD) | 2 years | |
| Median Time to ANC Engraftment | 100 days | |
| Median Time to Platelet Engraftment |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Shalini Shenoy, MD | Washington University School of Medicine | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Regents of University of California- UCLA | Los Angeles | California | 90095 | United States | ||
| Children's Hospital and Research Center at Oakland |
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Participants were recruited based on physician referral at 11 academic medical centers. The first participant was enrolled in May 2010 and the last participant was enrolled in April 2012.
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| ID | Title | Description |
|---|---|---|
| FG000 | Conditioning Regimen | Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan: Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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|
| 100 days |
| Incidence of Disease Recurrence | 2 years |
| Oakland |
| California |
| 94609 |
| United States |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | United States |
| University of Miami | Miami | Florida | 33136 | United States |
| All Children's Research Institute, Inc. | St. Petersburg | Florida | 33701 | United States |
| Emory University | Atlanta | Georgia | 30322 | United States |
| Children's Memorial Hospital | Chicago | Illinois | 60614 | United States |
| Dana Farber Cancer Institute | Boston | Massachusetts | 02115 | United States |
| University of Michigan | Ann Arbor | Michigan | 48109 | United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| The University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | United States |
| Duke University | Durham | North Carolina | 27710 | United States |
| The Research Institute at Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Oregon Health and Science University | Portland | Oregon | 97239 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Vanderbilt University | Nashville | Tennessee | 37232 | United States |
| UT Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| Methodist Healthcare System of San Antonio | San Antonio | Texas | 78229 | United States |
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Conditioning Regimen | Hydroxyurea days -50 to -21 Alemtuzumab days -21 to -19 Fludarabine days -8 to -4 Thiotepa day -4 Melphalan day -3 Stem cell infusion day 0 Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan: Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median | Full Range | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Count of Participants | 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 | Primary Objective: Event-free Survival at 1 Year. | Posted | Count of Participants | Participants | 1 year |
|
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| ||||||||||||||||||||||||||||
| Secondary | Development of Graft Versus Host Disease (GVHD) | Posted | Count of Participants | Participants | 2 years |
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| |||||||||||||||||||||||||||||
| Secondary | Median Time to ANC Engraftment | Posted | Median | Full Range | days | 100 days |
|
| ||||||||||||||||||||||||||||
| Secondary | Median Time to Platelet Engraftment | 3 participants did not engraft platelets | Posted | Median | Full Range | days | 100 days |
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| Secondary | Incidence of Disease Recurrence | Posted | Count of Participants | Participants | 2 years |
|
|
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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 | Conditioning Regimen | Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan: Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion | 4 | 21 | 10 | 21 | 21 | 21 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pulmonary toxicity | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | Respiratory distress, pulmonary failure, pulmonary hemorrhage |
| |
| Pain | General disorders | Systematic Assessment |
| ||
| Graft rejection | General disorders |
| |||
| Edema | General disorders | Head and neck edema |
| ||
| Posterior Reversible Encephalopathy Syndrome | Nervous system disorders |
| |||
| Sepsis | Infections and infestations | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| CMV reactivation | Infections and infestations | Systematic Assessment |
| ||
| Veno Occlusive Disease | Hepatobiliary disorders |
| |||
| Hyperbilirubinemia | Investigations |
| |||
| Hypercalcemia | Investigations |
| |||
| Acute Kidney Injury | Renal and urinary disorders |
| |||
| Infection (other than CMV) | Infections and infestations |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Shalini Shenoy | Washington University School of Medicine | 314-454-6018 | shalinishenoy@wustl.edu |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C024352 | fludarabine |
| D008558 | Melphalan |
| ID | Term |
|---|---|
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D010649 | Phenylalanine |
| D024322 | Amino Acids, Aromatic |
| D000598 | Amino Acids, Cyclic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
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| African American |
|
| Hispanic |
|
| Pakistani |
|
| Caucasian |
|
| Vietnamese |
|
| More than 1 race reported |
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| Title | Denominators | Categories | ||||
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