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| ID | Type | Description | Link |
|---|---|---|---|
| NA_00039363 | Other Identifier | JHM IRB |
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The stopping rule was met and hence the study was closed
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This research is being done to learn more about reduced-intensity bone marrow transplantation (BMT), also known as a "mini" transplant for patients with blood cancers, using bone marrow from a relative.
The main goal of the study is to determine how quickly the donor's bone marrow "takes" in your body. Other goals include describing how many people accept the bone marrow and how quickly the blood counts come up; describing Graft-versus-host disease (GVHD) and other complications; and describing how many people survive without progressive cancer and survive overall
At the present time there are few or no cures for people with cancer of the blood or lymph glands outside of a bone marrow transplant (BMT). BMT has developed over several decades of research as an effective treatment of various malignant and nonmalignant hematologic diseases.
This research is being done to learn more about reduced-intensity bone marrow transplantation (BMT), also known as a "mini" transplant for patients with blood cancers, using bone marrow from a relative. The bone marrow for this transplant comes from a relative who is a half-match or "haplo" match to you. Possible donors include parents, siblings, and children.
"Mini" transplants have been given to many people with various cancers but are considered experimental. Over 200 people at Johns Hopkins have received mini transplants with high doses of cyclophosphamide after the transplant. However, the chemotherapy combination and other treatment given before those transplants were different from what is in this study. Although all of the chemotherapy and immune-lowering drugs used in this study are approved by the Food and Drug Administration (FDA), the combination of medications used in this study are not FDA approved and are experimental.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BMT Allogenic Transplantation | Experimental | Reduced-intensity transplant with a fludarabine- and busulfan-based preparative regimen. GVHD prophylaxis with cyclophosphamide, tacrolimus, and mycophenolate mofetil. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Fludarabine | Drug | 30 mg/m^2 IV daily on Day -6 through Day -2. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Chimerism in Unsorted Peripheral Blood | Percentage of participants achieving full-donor chimerism in unsorted peripheral blood. | Day 60 |
| Chimerism in CD3+ Sorted Peripheral Blood | Percentage of participants achieving full-donor chimerism in CD3+ sorted peripheral blood | Day 60 |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Survival | Percentage of participants alive | 1 year |
| Progression-free Survival | Percentage of participants alive without disease relapse or progression. |
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Inclusion Criteria:
First-degree related donor who is at minimum HLA haploidentical
Eligible diagnoses:
Low-grade non-Hodgkin's lymphoma or plasma cell neoplasm that has progressed during multiagent therapy, failed at least two prior therapies (excluding single agent rituximab and single agent steroids), or in the case of lymphoma undergone histological conversion:
Poor-risk SLL or CLL, defined by an 11q or 17p deletion, histological conversion, or disease progression < 6 months after a purine analog-containing regimen
Aggressive lymphoma that has failed at least one prior regimen of multiagent chemotherapy, and patient is either ineligible for autologous BMT or autologous BMT is not recommended:
Relapsed or refractory acute leukemia in second or subsequent remission
Poor-risk acute leukemia in first remission
AML with at least one of the following:
AML arising from MDS or a myeloproliferative disorder, or secondary AML
Presence of Flt3 internal tandem duplications
Poor-risk cytogenetics
Primary refractory disease
Adverse cytogenetics
Clear evidence of hypodiploidy
Primary refractory disease
Interferon- or imatinib-refractory CML in first chronic phase, or non-blast crisis CML beyond first chronic phase
Philadelphia chromosome negative myeloproliferative disease (including myelofibrosis)
Chronic myelomonocytic leukemia
Juvenile myelomonocytic leukemia
Left ventricular ejection fraction greater than or equal to 35%
Bilirubin ≤ 3.0 mg/dL (unless due to Gilbert's syndrome or hemolysis), and ALT and AST < 5 x ULN
FEV1 and FVC > 40% of predicted; or in pediatric patients, if unable to perform pulmonary function tests due to young age, oxygen saturation >92% on room air
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Yvette Kasamon, M.D. | Johns Hopkins University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Sydney Kimmel Comprehensive Cancer Center | Baltimore | Maryland | 21231 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | BMT Allogenic Transplantation | Fludarabine, Busulfan, Cyclophosphamide, Tacrolimus,: Fludarabine 30 mg/m2 IV once a day for 4 days Busulfan 0.8 mg/kg IV every 12 hours for 4 days Cyclophosphamide 50 mg/kg IV daily for 2 days |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Busulfan | Drug | 1 mg/kg PO OR 0.8 mg/kg IV four times daily on Day -6 through Day -3. |
|
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| Cyclophosphamide | Drug | 50 mg/kg IV daily on Day +3 and Day +4. |
|
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| Mycophenolate Mofetil | Drug | 15 mg/kg PO three times daily (max daily dose of 3g) starting on Day +5. |
|
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| Tacrolimus | Drug | Dosed based on drug levels; begin on Day +5 at 1 mg IV daily. |
|
|
| 1 year |
| Incidence of Relapse | Percentage of participants experiencing disease relapse or progression | 1 year |
| Non-relapse Mortality | Percentage of participants who died due to BMT-related reasons | 1 year |
| Incidence of Graft-versus-host-disease (GVHD) | Percentage of participants experiencing acute and chronic GVHD. Acute GVHD is graded by Przepiorka criteria. Chronic GVHD is graded by NIH consensus criteria and Seattle criteria. | 1 year |
| Participants Who Failed to Engraft | Number of participants who failed to engraft | Day 60 |
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | BMT Allogenic Transplantation | Fludarabine, Busulfan, Cyclophosphamide, Tacrolimus,: Fludarabine 30 mg/m2 IV once a day for 4 days Busulfan 0.8 mg/kg IV every 12 hours for 4 days Cyclophosphamide 50 mg/kg IV daily for 2 days |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Full Range | years |
| ||||||||||||||||||||||
| Age, Categorical | Count of Participants | Participants |
| |||||||||||||||||||||||
| Sex/Gender, Customized | Number | 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 | Chimerism in Unsorted Peripheral Blood | Percentage of participants achieving full-donor chimerism in unsorted peripheral blood. | The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed. | Posted | Day 60 |
|
| ||||||||||||||||||||
| Primary | Chimerism in CD3+ Sorted Peripheral Blood | Percentage of participants achieving full-donor chimerism in CD3+ sorted peripheral blood | The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed. | Posted | Day 60 |
|
| ||||||||||||||||||||
| Secondary | Overall Survival | Percentage of participants alive | The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed. | Posted | 1 year |
|
| ||||||||||||||||||||
| Secondary | Progression-free Survival | Percentage of participants alive without disease relapse or progression. | The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed. | Posted | 1 year |
|
| ||||||||||||||||||||
| Secondary | Incidence of Relapse | Percentage of participants experiencing disease relapse or progression | The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed. | Posted | 1 year |
|
| ||||||||||||||||||||
| Secondary | Non-relapse Mortality | Percentage of participants who died due to BMT-related reasons | The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed. | Posted | 1 year |
|
| ||||||||||||||||||||
| Secondary | Incidence of Graft-versus-host-disease (GVHD) | Percentage of participants experiencing acute and chronic GVHD. Acute GVHD is graded by Przepiorka criteria. Chronic GVHD is graded by NIH consensus criteria and Seattle criteria. | The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed. | Posted | 1 year |
|
| ||||||||||||||||||||
| Secondary | Participants Who Failed to Engraft | Number of participants who failed to engraft | Posted | Count of Participants | Participants | Day 60 |
|
|
|
Up to 1 year
Data was collected systematically through at least Day 60 by investigator assessment at protocol-specified visits.
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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 | Reduced-intensity transplant with a fludarabine- and busulfan-based preparative regimen. GVHD prophylaxis with cyclophosphamide, tacrolimus, and mycophenolate mofetil. | 9 | 15 | 11 | 15 | 7 | 15 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Diarrhea | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Fever | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Chills | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| nausea | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| fever | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| tremor | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| hallucinations | Psychiatric disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Elevated liver enzymes | Investigations | CTCAE (4.0) | Systematic Assessment |
| |
| Elevated total bilirubin | Investigations | CTCAE (4.0) | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Febrile neutropenia | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Yvette Kasamon | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | 410-955-8839 | ykasamo1@jhmi.edu |
| ID | Term |
|---|---|
| D008223 | Lymphoma |
| D007938 | Leukemia |
| D009190 | Myelodysplastic Syndromes |
| D006689 | Hodgkin Disease |
| D008228 | Lymphoma, Non-Hodgkin |
| D015470 | Leukemia, Myeloid, Acute |
| D054198 | Precursor Cell Lymphoblastic Leukemia-Lymphoma |
| D015464 | Leukemia, Myelogenous, Chronic, BCR-ABL Positive |
| ID | Term |
|---|---|
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D006402 | Hematologic Diseases |
| D001855 | Bone Marrow Diseases |
| D007951 | Leukemia, Myeloid |
| D007945 | Leukemia, Lymphoid |
| D009196 | Myeloproliferative Disorders |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C024352 | fludarabine |
| C042382 | fludarabine phosphate |
| D007252 | Influenza Vaccines |
| D002066 | Busulfan |
| D003520 | Cyclophosphamide |
| D009173 | Mycophenolic Acid |
| D016559 | Tacrolimus |
| ID | Term |
|---|---|
| D014765 | Viral Vaccines |
| D014612 | Vaccines |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| 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 |
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D002208 | Caproates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D005227 | Fatty Acids |
| D008055 | Lipids |
| D018942 | Macrolides |
| D007783 | Lactones |
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| >=65 years |
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| Categories |
|---|
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