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Per protocol, the results of a planned interim analysis demonstrated insufficient efficacy and led to early termination of the study.
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| Name | Class |
|---|---|
| Novartis Pharmaceuticals | INDUSTRY |
Not provided
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This will be a single arm Phase II study.
LBH589 (20 mg PO) will be administered three times a week on Monday, Wednesday and Friday. Treatment will be given over 21 days followed by a 7 day rest period and repeated every 28 days. Patients will be assessed for toxicity on an ongoing basis and disease assessment will be determined every 2 treatment cycles (8 weeks). Patients will be allowed to continue on treatment for a maximum of eight four week treatment cycles. Treatment will be discontinued if there is evidence of disease progression, unacceptable toxicity and/or at the discretion of the investigator.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Panobinostat 20 mg | Experimental | Treatment with LBH589 (Panobinostat) 20 mg |
|
| Panobinostat 30 mg | Experimental | Treatment with LBH589 (Panobinostat) 30 mg |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Panobinostat | Drug | Panobinostat(20 mg or 30 mg PO) will be administered three times a week on Monday, Wednesday and Friday. Treatment will be given over 21 days followed by a 7 day rest period and repeated every 28 days. Patients will be assessed for toxicity on an ongoing basis and disease assessment will be determined every 2 treatment cycles (8 weeks). Patients will be allowed to continue on treatment for a maximum of eight four week treatment cycles. Treatment will be discontinued if there is evidence of disease progression, unacceptable toxicity and/or at the discretion of the investigator. |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Response Rate (CR, Marrow CR + PR) of LBH in Patients With Relapsed or Refractory MDS. | Overall response rate (ORR) is defined by the modified International Working Group (IWG) Response Criteria for MDS. In the marrow, Complete Response (CR) is <= 5% blasts present with normal maturation of all cell lines. In peripheral blood, CR is defined as hemoglobin >= 11 g/dL, ANC >= 1000/mL, and platelets >= 100,000 with 0% blasts present. Partial Response (PR) is defined the same as CR with blasts decreased by >= 50% and >= 5% blasts in the marrow. | Every 8 weeks up to 24 months on-study. |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Disease Progression | Time to disease progression is defined as the time between day 1 cycle 1 and time to first documented disease progression. Disease progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. |
Not provided
Inclusion Criteria:
Bilirubin <= 1.5 mg/dL AST/SGOT <= 2.5 x ULN ALT/SGPT Creatinine <= 2.0 mg/dL or 24-hour Creatinine Clearance >= 50 ml/min Albumin >= 3 g/dL Potassium >= lower limit normal (LLN) Phosphorous >= LLN Calcium >= LLN Magnesium >= LLN
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ian W. Flinn, M.D. | SCRI Development Innovations, LLC | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Florida Cancer Specialists | Fort Myers | Florida | 33901 | United States | ||
| Northeast Georgia Medical Center |
Not provided
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| ID | Title | Description |
|---|---|---|
| FG000 | Panobinostat 30 mg | Panobinostat(30 mg PO) will be administered three times a week on Monday, Wednesday and Friday. Treatment will be given over 21 days followed by a 7 day rest period and repeated every 28 days. Patients will be assessed for toxicity on an ongoing basis and disease assessment will be determined every 2 treatment cycles (8 weeks). Patients will be allowed to continue on treatment for a maximum of eight four week treatment cycles. Treatment will be discontinued if there is evidence of disease progression, unacceptable toxicity and/or at the discretion of the investigator. |
| FG001 | Panobinostat 20 mg | Panobinostat(20 mg PO) will be administered three times a week on Monday, Wednesday and Friday. Treatment will be given over 21 days followed by a 7 day rest period and repeated every 28 days. Patients will be assessed for toxicity on an ongoing basis and disease assessment will be determined every 2 treatment cycles (8 weeks). Patients will be allowed to continue on treatment for a maximum of eight four week treatment cycles. Treatment will be discontinued if there is evidence of disease progression, unacceptable toxicity and/or at the discretion of the investigator. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Panobinostat 30 mg | Panobinostat(30 mg PO) will be administered three times a week on Monday, Wednesday and Friday. Treatment will be given over 21 days followed by a 7 day rest period and repeated every 28 days. Patients will be assessed for toxicity on an ongoing basis and disease assessment will be determined every 2 treatment cycles (8 weeks). Patients will be allowed to continue on treatment for a maximum of eight four week treatment cycles. Treatment will be discontinued if there is evidence of disease progression, unacceptable toxicity and/or at the discretion of the investigator. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median |
| 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 | Overall Response Rate (CR, Marrow CR + PR) of LBH in Patients With Relapsed or Refractory MDS. | Overall response rate (ORR) is defined by the modified International Working Group (IWG) Response Criteria for MDS. In the marrow, Complete Response (CR) is <= 5% blasts present with normal maturation of all cell lines. In peripheral blood, CR is defined as hemoglobin >= 11 g/dL, ANC >= 1000/mL, and platelets >= 100,000 with 0% blasts present. Partial Response (PR) is defined the same as CR with blasts decreased by >= 50% and >= 5% blasts in the marrow. | All evaluable patients assessed for response prior to early study termination - one patient in each arm was not evaluable due to coming off-study prior to assessment | Posted | Number | participants | Every 8 weeks up to 24 months on-study. |
|
Not provided
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Panobinostat 20 mg | Panobinostat(20 mg PO) will be administered three times a week on Monday, Wednesday and Friday. Treatment will be given over 21 days followed by a 7 day rest period and repeated every 28 days. Patients will be assessed for toxicity on an ongoing basis and disease assessment will be determined every 2 treatment cycles (8 weeks). Patients will be allowed to continue on treatment for a maximum of eight four week treatment cycles. Treatment will be discontinued if there is evidence of disease progression, unacceptable toxicity and/or at the discretion of the investigator. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal Pain | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Platelet Count Decreased | Investigations | CTCAE (4.0) | Systematic Assessment |
Results of a planned interim analysis demonstrated that the regimen did not demonstrate sufficient evidence of efficacy necessary to justify further enrollment. Per protocol, the results of this analysis led to early termination of the study.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| John Hainsworth, MD | Sarah Cannon Research Institute | 1-877-691-7274 | ASKSARAH@scresearch.net |
Not provided
| ID | Term |
|---|---|
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D000077767 | Panobinostat |
| ID | Term |
|---|---|
| D006877 | Hydroxamic Acids |
| D006898 | Hydroxylamines |
| D000588 | Amines |
| D009930 | Organic Chemicals |
Not provided
Not provided
Not provided
Not provided
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Not provided
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|
|
| Every 8 weeks up to 24 months on-study, every 3 months in follow-up until progression of disease |
| Hematologic Improvement, Including Transfusion Independence | Hematologic measures will include total WBC and platelets | Every 8 weeks up to 24 months on-study |
| Duration of Response | Duration of response is defined as the time from when objective response is realized until time to first documented disease progression. Disease progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Objective Response = CR + PR. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI or CT: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions. | Every 8 weeks up to 24 months on-study |
| Median Time to Treatment Failure | Time to treatment failure is defined as measuring the time between cycle 1 day 1 to discontinuation for any reason. | 24 months |
| Median Overall Survival | The Length of Time, in Months, That Patients Were Alive From Their First Date of Protocol Treatment Until Death | 24 months on-study, patients followed every 3 months in follow-up |
| Safety and Tolerability of LBH589 in Patients With Relapsed/Refractory MDS by Measuring the Number of Participants With Adverse Events | The reported incidence of AEs and SAEs with an onset on or after the initiation of therapy was graded according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0 | 24 months |
| Gainesville |
| Georgia |
| 30501 |
| United States |
| Consultants in Blood Disorders and Cancer | Louisville | Kentucky | 40207 | United States |
| Center for Cancer and Blood Disorders | Bethesda | Maryland | 20817 | United States |
| Oncology Hematology Care | Cincinnati | Ohio | 45242 | United States |
| Chattanooga Oncology Hematology Associates | Chattanooga | Tennessee | 37404 | United States |
| Tennessee Oncology, PLLC | Nashville | Tennessee | 37023 | United States |
| BG001 | Panobinostat 20 mg | Panobinostat(20 mg PO) will be administered three times a week on Monday, Wednesday and Friday. Treatment will be given over 21 days followed by a 7 day rest period and repeated every 28 days. Patients will be assessed for toxicity on an ongoing basis and disease assessment will be determined every 2 treatment cycles (8 weeks). Patients will be allowed to continue on treatment for a maximum of eight four week treatment cycles. Treatment will be discontinued if there is evidence of disease progression, unacceptable toxicity and/or at the discretion of the investigator. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| OG001 | Panobinostat 20 mg | Panobinostat(20 mg PO) will be administered three times a week on Monday, Wednesday and Friday. Treatment will be given over 21 days followed by a 7 day rest period and repeated every 28 days. Patients will be assessed for toxicity on an ongoing basis and disease assessment will be determined every 2 treatment cycles (8 weeks). Patients will be allowed to continue on treatment for a maximum of eight four week treatment cycles. Treatment will be discontinued if there is evidence of disease progression, unacceptable toxicity and/or at the discretion of the investigator. |
|
|
| Secondary | Time to Disease Progression | Time to disease progression is defined as the time between day 1 cycle 1 and time to first documented disease progression. Disease progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. | Study terminated early, no results are available for this endpoint as the analysis was not done. | Posted | Every 8 weeks up to 24 months on-study, every 3 months in follow-up until progression of disease |
|
|
| Secondary | Hematologic Improvement, Including Transfusion Independence | Hematologic measures will include total WBC and platelets | Study terminated early, no results are available for this endpoint as the analysis was not done | Posted | Every 8 weeks up to 24 months on-study |
|
|
| Secondary | Duration of Response | Duration of response is defined as the time from when objective response is realized until time to first documented disease progression. Disease progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Objective Response = CR + PR. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI or CT: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions. | Study terminated early, no results are available for this endpoint as the analysis was not done | Posted | Every 8 weeks up to 24 months on-study |
|
|
| Secondary | Median Time to Treatment Failure | Time to treatment failure is defined as measuring the time between cycle 1 day 1 to discontinuation for any reason. | Study terminated early, no results are available for this endpoint as the analysis was not done | Posted | 24 months |
|
|
| Secondary | Median Overall Survival | The Length of Time, in Months, That Patients Were Alive From Their First Date of Protocol Treatment Until Death | Study terminated early, no results are available for this endpoint as the analysis was not done | Posted | 24 months on-study, patients followed every 3 months in follow-up |
|
|
| Secondary | Safety and Tolerability of LBH589 in Patients With Relapsed/Refractory MDS by Measuring the Number of Participants With Adverse Events | The reported incidence of AEs and SAEs with an onset on or after the initiation of therapy was graded according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0 | Posted | Count of Participants | Participants | 24 months |
|
|
|
| 3 |
| 10 |
| 9 |
| 10 |
| EG001 | Panobinostat 30 mg | Panobinostat(30 mg PO) will be administered three times a week on Monday, Wednesday and Friday. Treatment will be given over 21 days followed by a 7 day rest period and repeated every 28 days. Patients will be assessed for toxicity on an ongoing basis and disease assessment will be determined every 2 treatment cycles (8 weeks). Patients will be allowed to continue on treatment for a maximum of eight four week treatment cycles. Treatment will be discontinued if there is evidence of disease progression, unacceptable toxicity and/or at the discretion of the investigator. | 6 | 16 | 15 | 16 |
| Febrile neutropenia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
|
| Infections and infestations - Other, pneumonia | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
|
| Platelet count decreased | Investigations | CTCAE (4.0) | Systematic Assessment |
|
| Pain | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| General disorders and administration site conditions - Other, compartment syndrome | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Infections and infestations - Other, unspecified | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
|
| Atrial Fibrillation | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
|
| Anemia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
|
| Anemia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
|
| Fatigue | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Neutrophil Count Decreased | Investigations | CTCAE (4.0) | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Anorexia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Dyspnea | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | CTCAE (4.0) | Systematic Assessment |
|
| White Blood Cell Decreased | Investigations | CTCAE (4.0) | Systematic Assessment |
|
| Dizziness | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
|
| Edema | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Generalized Muscle Weakness | Musculoskeletal and connective tissue disorders | CTCAE (4.0) | Systematic Assessment |
|
| Peripheral Sensory Neuropathy | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Fever | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Gastroesophageal Reflux Disease | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Dysgeusia | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
|
| Gastrointestinal Disorders - Other, Stomatitis | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Non-Cardiac Chest Pain | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | CTCAE (4.0) | Systematic Assessment |
|
| Abdominal Pain | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Bruising | Injury, poisoning and procedural complications | CTCAE (4.0) | Systematic Assessment |
|
| Cardiac Disorders - Other, Systolic Murmur | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
|
| Depression | Psychiatric disorders | CTCAE (4.0) | Systematic Assessment |
|
| Electrocardiogram Qt Corrected Interval Prolonged | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| General Disorders And Administration Site Conditions - Other, Pain At Port Site | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Hypomagnesemia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
|
| Skin And Subcutaneous Tissue Disorders - Other, Ecchymosis | Skin and subcutaneous tissue disorders | CTCAE (4.0) | Systematic Assessment |
|
| Cardiac Disorders - Other, Tachycardia | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
|
| Dry Skin | Skin and subcutaneous tissue disorders | CTCAE (4.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
|
| Hematoma | Vascular disorders | CTCAE (4.0) | Systematic Assessment |
|
| Hyperthyroidism | Endocrine disorders | CTCAE (4.0) | Systematic Assessment |
|
| Hypokalemia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
|
| Hypotension | Vascular disorders | CTCAE (4.0) | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | CTCAE (4.0) | Systematic Assessment |
|
| Respiratory, Thoracic And Mediastinal Disorders - Other, Hemoptysis | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Skin And Subcutaneous Tissue Disorders - Other, Petechiae | Skin and subcutaneous tissue disorders | CTCAE (4.0) | Systematic Assessment |
|
| Skin Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
|
| Upper Respiratory Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
|
| Urinary Frequency | Renal and urinary disorders | CTCAE (4.0) | Systematic Assessment |
|
| Weight Loss | Investigations | CTCAE (4.0) | Systematic Assessment |
|
The sponsor can review/embargo results communications prior to public release for a period that is >60 days but ≤180 days from date submitted to sponsor, who may require changes to the communication in order to remove specifically identified confidential information (other than study data) and/or delay the proposed publication to enable the sponsor to seek patent protection for inventions. The PI may not publish its results until 18 mos. after the trial has been completed at all sites.
| D006880 |
| Hydroxy Acids |
| D002264 | Carboxylic Acids |
| D007211 | Indoles |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |