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| ID | Type | Description | Link |
|---|---|---|---|
| ELT115895 | Other Identifier | Novartis |
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| Name | Class |
|---|---|
| Novartis | INDUSTRY |
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The investigators hypothesis is that eltrombopag given to patients with moderate to very severe aplastic anemia will result in an increase in platelet counts. The investigators hypothesize that in patients with moderate to very severe aplastic anemia, treatment with eltrombopag will lead to fewer platelet transfusions, red blood cell transfusions, and fewer bleeding events. The investigators hypothesize that in patients with moderate to very severe aplastic anemia, eltrombopag will have an acceptable toxicity rate <3%, at doses that result in increased platelet counts. Finally the investigators hypothesize that plasma eltrombopag levels in peripheral blood will correlate with improved platelet counts.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eltrombopag | Experimental | Single arm study. Dose Escalation. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eltrombopag | Drug | Oral eltrombopag 150mg/day by mouth starting on Day 1 with dose modification over 12 weeks to a maximum of 300mg/day determined by platelet count |
|
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Participants With Platelet Response | Defined as a stable platelet count of 50,000/μl or more during any 4 week period within the possible 12 weeks while on study,and including maximal platelet counts achieved in patients with moderate to very severe aplastic anemia. | up to 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Platelet Count Twice Baseline. | Proportion of subjects who achieve platelet counts at least twice their baseline value at any point while on study medication, in patients with moderate to very severe aplastic anemia. | Between weeks 1-12. |
| Hematology Labs |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| George M Rodgers, M.D. | University of Utah | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Utah | Salt Lake City | Utah | 84112 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Eltrombopag | Single arm study. Dose Escalation. Eltrombopag: Oral eltrombopag 150mg/day by mouth starting on Day 1 with dose modification over 12 weeks to a maximum of 300mg/day determined by platelet count |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Eltrombopag | Single arm study. Dose Escalation. Eltrombopag: Oral eltrombopag 150mg/day by mouth starting on Day 1 with dose modification over 12 weeks to a maximum of 300mg/day determined by platelet count |
| 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 |
| 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 | Proportion of Participants With Platelet Response | Defined as a stable platelet count of 50,000/μl or more during any 4 week period within the possible 12 weeks while on study,and including maximal platelet counts achieved in patients with moderate to very severe aplastic anemia. | Posted | Number | 95% Confidence Interval | proportion of participants | up to 12 weeks |
|
|
Approximately 3 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 | Eltrombopag | Single arm study. Dose Escalation. Eltrombopag: Oral eltrombopag 150mg/day by mouth starting on Day 1 with dose modification over 12 weeks to a maximum of 300mg/day determined by platelet count |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| congestive heart failure | Cardiac disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| abdominal pain | Gastrointestinal disorders | Systematic Assessment |
Certain data regarding the primary outcome and adverse events were inadvertently not captured during the clinical investigation resulting in insufficient data for statistical analysis and publication.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Kimberlee Taylor | Huntsman Cancer Institute | 8012135673 | Kimberlee.Taylor@hci.utah.edu |
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| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| D006470 | Hemorrhage |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
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| ID | Term |
|---|---|
| C520809 | eltrombopag |
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Association between eltrombopag use and response in hemoglobin, hematocrit, total white blood cell count, and absolute neutrophil count to be evaluate by maximal hemoglobin, hematocrit, total white blood cell count, and absolute neutrophil counts achieved in patients with moderate to very severe aplastic anemia |
| 12 weeks |
| Number of Patients With AE to Measure Toxicity, Using NCI CTCAE | Evaluated weekly, up to 12 weeks. Association between eltrombopag use, dose, and tolerability in patients with moderate to very severe aplastic anemia | 12 weeks |
| Characterization of the PK Profile of Eltrombopag in Patients With Moderate to Very Severe Aplastic Anemia. Evaluated With AUC, Cmax, Cmin, Tmax. | Samples will for PK analysis will collected as a trough level weeks 2, 6 and 12, prior to dose of eltrombopag. Additional PK level drawn at 2, 4 and 6 hours post-dose at the scheduled week 2 visit. | Weeks 2, 6 and 12 |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | Count of Participants |
|
|
|
|
| Secondary | Platelet Count Twice Baseline. | Proportion of subjects who achieve platelet counts at least twice their baseline value at any point while on study medication, in patients with moderate to very severe aplastic anemia. | Data was not collected for this outcome variable. | Posted | Between weeks 1-12. |
|
|
| Secondary | Hematology Labs | Association between eltrombopag use and response in hemoglobin, hematocrit, total white blood cell count, and absolute neutrophil count to be evaluate by maximal hemoglobin, hematocrit, total white blood cell count, and absolute neutrophil counts achieved in patients with moderate to very severe aplastic anemia | Data was not collected for this outcome variable. | Posted | 12 weeks |
|
|
| Secondary | Number of Patients With AE to Measure Toxicity, Using NCI CTCAE | Evaluated weekly, up to 12 weeks. Association between eltrombopag use, dose, and tolerability in patients with moderate to very severe aplastic anemia | Data was not collected for this outcome variable. | Posted | 12 weeks |
|
|
| Secondary | Characterization of the PK Profile of Eltrombopag in Patients With Moderate to Very Severe Aplastic Anemia. Evaluated With AUC, Cmax, Cmin, Tmax. | Samples will for PK analysis will collected as a trough level weeks 2, 6 and 12, prior to dose of eltrombopag. Additional PK level drawn at 2, 4 and 6 hours post-dose at the scheduled week 2 visit. | Data was not collected for this outcome variable. | Posted | Weeks 2, 6 and 12 |
|
|
| 2 |
| 13 |
| 3 |
| 13 |
| 13 |
| 13 |
| cholecystitis | Hepatobiliary disorders | Systematic Assessment |
|
| syncope | Nervous system disorders | Systematic Assessment |
|
| aches/pains | General disorders | Systematic Assessment |
|
| anemia | Blood and lymphatic system disorders | Systematic Assessment |
|
| anxiety | Psychiatric disorders | Systematic Assessment |
|
| arrhythmia | Cardiac disorders | Systematic Assessment |
|
| back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| purpura | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| bronzing of the skin | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| bruising | Injury, poisoning and procedural complications | Systematic Assessment |
|
| chest pain | Cardiac disorders | Systematic Assessment |
|
| chills | General disorders | Systematic Assessment |
|
| confusion | Psychiatric disorders | Systematic Assessment |
|
| constipation | Gastrointestinal disorders | Systematic Assessment |
|
| cramps | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| decreased lymphocyte count | Investigations | Systematic Assessment |
|
| decreased neutrophil count | Investigations | Systematic Assessment |
|
| thrombocytopenia | Investigations | Systematic Assessment |
|
| dehydration | Metabolism and nutrition disorders | Systematic Assessment |
|
| diarrhea | Gastrointestinal disorders | Systematic Assessment |
|
| dizziness | Nervous system disorders | Systematic Assessment |
|
| dry mouth | Gastrointestinal disorders | Systematic Assessment |
|
| dry skin | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| dysgeusia | Nervous system disorders | Systematic Assessment |
|
| dysuria | Renal and urinary disorders | Systematic Assessment |
|
| ecchymosis | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| edema | General disorders | Systematic Assessment |
|
| Alkaline phosphatase increased | Investigations | Systematic Assessment |
|
| elevated ALT | Investigations | Systematic Assessment |
|
| elevated AST | Investigations | Systematic Assessment |
|
| elevated bilirubin | Investigations | Systematic Assessment |
|
| elevated creatinine | Investigations | Systematic Assessment |
|
| hyperglycemia | Metabolism and nutrition disorders | Systematic Assessment |
|
| epistaxis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| erythema | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| fatigue | General disorders | Systematic Assessment |
|
| gum hypertrophy | Infections and infestations | Systematic Assessment |
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| gum lesions | Infections and infestations | Systematic Assessment |
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| facial hair growth | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| headache | Nervous system disorders | Systematic Assessment |
|
| hematoma | Vascular disorders | Systematic Assessment |
|
| hot flashes | Vascular disorders | Systematic Assessment |
|
| hypertension | Vascular disorders | Systematic Assessment |
|
| hypoalbunemia | Investigations | Systematic Assessment |
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| hypocalcemia | Metabolism and nutrition disorders | Systematic Assessment |
|
| hyponatremia | Metabolism and nutrition disorders | Systematic Assessment |
|
| hypoxia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| insomnia | Psychiatric disorders | Systematic Assessment |
|
| jaundice | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| menorrhagia | Reproductive system and breast disorders | Systematic Assessment |
|
| mouth sores | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| nasal congestion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| otitis externa | Infections and infestations | Systematic Assessment |
|
| nausea | Gastrointestinal disorders | Systematic Assessment |
|
| night sweats | General disorders | Systematic Assessment |
|
| opacity | Eye disorders | Systematic Assessment |
|
| pain | General disorders | Systematic Assessment |
|
| pallor | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| petechiae | Blood and lymphatic system disorders | Systematic Assessment |
|
| port leak | General disorders | Systematic Assessment |
|
| pyelonephritis | Renal and urinary disorders | Systematic Assessment |
|
| rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| rectal hemorrhage | Gastrointestinal disorders | Systematic Assessment |
|
| renal insufficiency | Renal and urinary disorders | Systematic Assessment |
|
| respiratory infection | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| sciatica | Nervous system disorders | Systematic Assessment |
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| scleral icterus | Eye disorders | Systematic Assessment |
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| shortness of breath | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| spelnomegaly | Hepatobiliary disorders | Systematic Assessment |
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| thrush | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| transfusion reaction | Blood and lymphatic system disorders | Systematic Assessment |
|
| tremor | Nervous system disorders | Systematic Assessment |
|
| urinary tract infection | Infections and infestations | Systematic Assessment |
|
| weight gain | Investigations | Systematic Assessment |
|
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| D001855 | Bone Marrow Diseases |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |