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The purpose of this open-label, single arm, multi-center Expanded Treatment Protocol (ETP) was to provide early access to ruxolitinib and evaluate safety information in patients with polycythemia vera (PV) who were hydroxyurea (HU) resistant or intolerant and who had no other standard treatment option, nor did they qualify for another clinical study for PV
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All patients | Experimental | All patients will receive ruxolitinib at a starting dose of 10 mg twice daily which could be titrated to most appropriate dose. Dose was not to exceed 25 mg bid nor be less than 5 mg once a day |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ruxolitinib | Drug | supplied as 5 mg, 10 mg and 20 mg tablets to be taken orally |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events - All Grades | Summary of adverse events (all grades). | Baseline up to approximately 26 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Hematocrit Levels at All Visits | Change in hematocrit levels from Baseline to each visit were measured | Up to approximately 26 months |
| Change From Baseline in Spleen Length |
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Inclusion Criteria:
•Confirmed diagnosis of PV according to the 2008 World Health Organization criteria, palpable spleen, Resistant to or intolerant of hydroxyurea, ECOG performance status of 0, 1 or 2; did not have access to a comparable or satisfactory alternative treatment
Exclusion Criteria:
•Inadequate liver or renal function, Significant bacterial, fungal, parasitic, or viral infection requiring treatment, Active malignancy within the past 5 years, except treated cervical intraepithelial neoplasia, basal cell carcinoma of the skin, or squamous cell carcinoma of the skin, with no evidence for recurrence in the past 3 years., Women who were pregnant or nursing.
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Linz | A-4010 | Austria | |||
| Novartis Investigative Site |
161 patients were included in the ruxolitinib arm and 100% of the patient's received the treatment
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| ID | Title | Description |
|---|---|---|
| FG000 | All Patients | All patients will receive ruxolitinib at a starting dose of 10 mg twice daily which could be titrated to most appropriate dose. Dose was not to exceed 25 mg bid nor be less than 5 mg once a day |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 11, 2016 | Dec 19, 2018 |
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Change in spleen length from Baseline to each visit
| Up to approximately 26 months |
| Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) | The MPN-SAF (Appendix 6) was a disease specific questionnaire comprised of 10 items that measures fatigue related to MPN disease and the severity of nine of the most prevalent associated symptoms including: early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, bone pain, fever and weight loss. There were three recall periods used in this questionnaire, which were 24 hours for fatigue, the past week for symptoms of early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, bone pain and fever, and the past 6 months for weight loss, Each item was scored on a scale ranging from 0 (no fatigue/absent) to 10 (As bad as you can imagine/worst imaginable). The MPN-SAF TSS was computed as the average of the observed items multiplied by 10 to achieve a 0-to-100 scale. The MPN-SAF TSS thus had a possible score range of 0 to 100. | Up to approximately 26 months |
| Salzburg |
| 5020 |
| Austria |
| Novartis Investigative Site | Wels | A 4600 | Austria |
| Novartis Investigative Site | Antwerp | 2060 | Belgium |
| Novartis Investigative Site | Bruges | 8000 | Belgium |
| Novartis Investigative Site | Brussels | 1070 | Belgium |
| Novartis Investigative Site | Leuven | 3000 | Belgium |
| Novartis Investigative Site | Liège | 4000 | Belgium |
| Novartis Investigative Site | Yvoir | 5530 | Belgium |
| Novartis Investigative Site | Pleven | 5800 | Bulgaria |
| Novartis Investigative Site | Plovdiv | 4002 | Bulgaria |
| Novartis Investigative Site | Sofia | 1413 | Bulgaria |
| Novartis Investigative Site | Sofia | 1431 | Bulgaria |
| Novartis Investigative Site | Vancouver | British Columbia | V5Z 1M9 | Canada |
| Novartis Investigative Site | Hamilton | Ontario | L8V 5C2 | Canada |
| Novartis Investigative Site | Viña del Mar | Valparaiso | 2540364 | Chile |
| Novartis Investigative Site | Santiago | 8420383 | Chile |
| Novartis Investigative Site | Santiago | Chile |
| Novartis Investigative Site | Bayonne | Bayonne Cedex | 64109 | France |
| Novartis Investigative Site | Le Mans | Cedex 09 | 72037 | France |
| Novartis Investigative Site | Angers | 49033 | France |
| Novartis Investigative Site | Avignon | 84902 | France |
| Novartis Investigative Site | Bordeaux | 33076 | France |
| Novartis Investigative Site | Chambéry | 73011 | France |
| Novartis Investigative Site | Marseille | 13273 | France |
| Novartis Investigative Site | Meaux | 77104 | France |
| Novartis Investigative Site | Metz | 57000 | France |
| Novartis Investigative Site | Mulhouse | 68070 | France |
| Novartis Investigative Site | Nice | 06202 | France |
| Novartis Investigative Site | Paris | 75010 | France |
| Novartis Investigative Site | Perpignan | 66046 | France |
| Novartis Investigative Site | Pringy | 74374 | France |
| Novartis Investigative Site | Toulouse | 31059 | France |
| Novartis Investigative Site | Vandœuvre-lès-Nancy | 54511 | France |
| Novartis Investigative Site | Villejuif | 94805 | France |
| Novartis Investigative Site | Mannheim | Baden-Wurttemberg | 68305 | Germany |
| Novartis Investigative Site | Aschaffenburg | 63739 | Germany |
| Novartis Investigative Site | Augsburg | 86150 | Germany |
| Novartis Investigative Site | Bad Soden | 65812 | Germany |
| Novartis Investigative Site | Berlin | 13357 | Germany |
| Novartis Investigative Site | Bottrop | 46236 | Germany |
| Novartis Investigative Site | Eisenach | 99817 | Germany |
| Novartis Investigative Site | Erlangen | 91054 | Germany |
| Novartis Investigative Site | Frankfurt | 60389 | Germany |
| Novartis Investigative Site | Frankfurt | 60596 | Germany |
| Novartis Investigative Site | Friedrichshafen | 88045 | Germany |
| Novartis Investigative Site | Hamburg | 22081 | Germany |
| Novartis Investigative Site | Hamm | 59063 | Germany |
| Novartis Investigative Site | Heidelberg | 69115 | Germany |
| Novartis Investigative Site | Heilbronn | 74072 | Germany |
| Novartis Investigative Site | Koblenz | 56068 | Germany |
| Novartis Investigative Site | Mutlangen | 73557 | Germany |
| Novartis Investigative Site | Stuttgart | 70376 | Germany |
| Novartis Investigative Site | Würzburg | 97080 | Germany |
| Novartis Investigative Site | Monterrey | Nuevo León | 64000 | Mexico |
| Novartis Investigative Site | Fredrikstad | NO-1603 | Norway |
| Novartis Investigative Site | Tromsø | 9038 | Norway |
| Novartis Investigative Site | Lisbon | 1099 023 | Portugal |
| Novartis Investigative Site | Lisbon | 1749-035 | Portugal |
| Novartis Investigative Site | Luleå | SE 971 80 | Sweden |
| Novartis Investigative Site | Uddevalla | 451 80 | Sweden |
| Novartis Investigative Site | Khon Kaen | THA | 40002 | Thailand |
| Novartis Investigative Site | Bangkok | 10400 | Thailand |
| Novartis Investigative Site | Bangkok | 10700 | Thailand |
| Novartis Investigative Site | Chiang Mai | 50200 | Thailand |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | All Patients | All patients will receive ruxolitinib at a starting dose of 10 mg twice daily which could be titrated to most appropriate dose. Dose was not to exceed 25 mg bid nor be less than 5 mg once a day |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Number | participants |
| |||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Number | participants |
| |||||||||||||||||||||||
| Number of participants resistant or intolerant to hydroxyurea (HU) | Definition of resistant or intolerant was based on physician's evaluation | Number | participants |
| ||||||||||||||||||||||
| Number of participants' frequency of phlebotomy in 52 weeks prior to screening | Number | participants |
| |||||||||||||||||||||||
| Number of participants' use of prior antineoplastic therapy | Other prior anti-neoplastic medications included busulfan, interferon, peginterferon, pipobroman, anagrelide, momelotinib, tioguanine, mercapturine and sodium phosphate | 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 | Number of Participants With Adverse Events - All Grades | Summary of adverse events (all grades). | Posted | Count of Participants | Participants | Baseline up to approximately 26 months |
|
|
| ||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Hematocrit Levels at All Visits | Change in hematocrit levels from Baseline to each visit were measured | Number of participants qualifying for evaluation of change from baseline varied across visits | Posted | Mean | Standard Deviation | percentage | Up to approximately 26 months |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Spleen Length | Change in spleen length from Baseline to each visit | Number of participants qualifying for evaluation of change from baseline varied across visits | Posted | Mean | Standard Deviation | cm | Up to approximately 26 months |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) | The MPN-SAF (Appendix 6) was a disease specific questionnaire comprised of 10 items that measures fatigue related to MPN disease and the severity of nine of the most prevalent associated symptoms including: early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, bone pain, fever and weight loss. There were three recall periods used in this questionnaire, which were 24 hours for fatigue, the past week for symptoms of early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, bone pain and fever, and the past 6 months for weight loss, Each item was scored on a scale ranging from 0 (no fatigue/absent) to 10 (As bad as you can imagine/worst imaginable). The MPN-SAF TSS was computed as the average of the observed items multiplied by 10 to achieve a 0-to-100 scale. The MPN-SAF TSS thus had a possible score range of 0 to 100. | Number of participants qualifying for evaluation of change from baseline varied across visits | Posted | Mean | Standard Deviation | scores | Up to approximately 26 months |
|
Adverse Events (AEs) were collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit) up to approximately 26 months
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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 | All Patients | All patients will receive ruxolitinib at a starting dose of 10 mg twice daily which could be titrated to most appropriate dose. Dose was not to exceed 25 mg bid nor be less than 5 mg once a day | 1 | 161 | 26 | 161 | 107 | 161 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Bundle branch block bilateral | Cardiac disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Pericarditis | Cardiac disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Ventricular extrasystoles | Cardiac disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Ascites | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Retroperitoneal haematoma | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Umbilical hernia | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Prostatitis Escherichia coli | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Accident | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
| |
| Acetabulum fracture | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
| |
| Overdose | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
| |
| Skin injury | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
| |
| Tendon rupture | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Acute myeloid leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.0) | Systematic Assessment |
| |
| Malignant melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.0) | Systematic Assessment |
| |
| Renal cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.0) | Systematic Assessment |
| |
| Squamous cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Thrombocytosis | Blood and lymphatic system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA (20.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (20.0) | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novaratis Pharmaceuticals | 888-669-6682 | novartis.email@novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 15, 2018 | Dec 19, 2018 | SAP_001.pdf |
| ID | Term |
|---|---|
| D011087 | Polycythemia Vera |
| D006402 | Hematologic Diseases |
| D009196 | Myeloproliferative Disorders |
| ID | Term |
|---|---|
| D019046 | Bone Marrow Neoplasms |
| D019337 | Hematologic Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D001855 | Bone Marrow Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C540383 | ruxolitinib |
Not provided
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Not provided
| Other |
|
| >=3 phlebotomies |
|
| Missing |
|
|
|
Change from baseline |
|
|