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Extend evaluation of deferasirox film-coated tablet (FCT) formulation
Collection of additional safety and efficacy data with deferasirox film-coated tablet (FCT) in patients who had completed study CICL670F2201
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Deferasirox | Experimental | Treatment will be administered daily for up to 24 months. For each patient the daily dose is calculated based on the patient's actual body weight. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deferasirox | Drug | Deferasirox FCT will be provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Overview of Number of Participants With Adverse Events | Numbers represent counts of participants within the categories. An adverse event (AE) was defined as treatment emergent if its onset date is on or after (≥) the first administration of study treatment within this study or events present prior to start of study treatment but increased in severity on or after (≥) the first administration of study treatment within this study but not later than 30 days after the last study treatment in this study | Baseline up to approximately 25 months |
| Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months |
| Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months |
| Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months |
| Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline. A negative change from baseline is regarded as an improvement in this study | Baseline, 6 and 12 months |
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Key Inclusion Criteria for subjects:
Key Exclusion for subjects:
The exclusion criteria followed those described for the core protocol CICl670F2201, which were as follows:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Vienna | 1140 | Austria | |||
| Novartis Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32793403 | Derived | Tartaglione I, Origa R, Kattamis A, Pfeilstocker M, Gunes S, Crowe S, Fagan N, Vincenzi B, Ruffo GB. Two-year long safety and efficacy of deferasirox film-coated tablets in patients with thalassemia or lower/intermediate risk MDS: phase 3 results from a subset of patients previously treated with deferasirox in the ECLIPSE study. Exp Hematol Oncol. 2020 Aug 10;9:20. doi: 10.1186/s40164-020-00174-2. eCollection 2020. |
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Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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All patients completed study CICL670F2201 (NCT02125877) prior to entry into this study
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| ID | Title | Description |
|---|---|---|
| FG000 | Deferasirox | Treatment will be administered daily for up to 24 months. For each patient the daily dose is calculated based on the patient's actual body weight. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Deferasirox | Treatment will be administered daily for up to 24 months. For each patient the daily dose is calculated based on the patient's actual body weight. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Number |
| 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 | Overview of Number of Participants With Adverse Events | Numbers represent counts of participants within the categories. An adverse event (AE) was defined as treatment emergent if its onset date is on or after (≥) the first administration of study treatment within this study or events present prior to start of study treatment but increased in severity on or after (≥) the first administration of study treatment within this study but not later than 30 days after the last study treatment in this study | Safety analysis set | Posted | Number | number of participants | Baseline up to approximately 25 months |
|
Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
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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 | Deferasirox | Treatment will be administered daily for up to 24 months. For each patient the daily dose is calculated based on the patient's actual body weight |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial fibrillation | Cardiac disorders | MedDRA (22.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Palpitations | Cardiac disorders | MedDRA (22.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 888-778-8300 | Novartis.email@novartis.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 26, 2019 | Jan 22, 2020 | SAP_000.pdf |
| Prot | Yes | No | No | Study Protocol | Oct 25, 2017 | Jan 22, 2020 | Prot_001.pdf |
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| ID | Term |
|---|---|
| D000740 | Anemia |
| D009190 | Myelodysplastic Syndromes |
| D013789 | Thalassemia |
| D019190 | Iron Overload |
| ID | Term |
|---|---|
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D001855 | Bone Marrow Diseases |
| D000745 | Anemia, Hemolytic, Congenital |
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| ID | Term |
|---|---|
| D000077588 | Deferasirox |
| ID | Term |
|---|---|
| D001565 | Benzoates |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
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| Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months |
| Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months |
| Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months |
| Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12 | The percentage relative change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point. A negative percentage relative change from baseline is regarded as an improvement in this study | Baseline, 6 and 12 months |
| Goudi-Athens |
| GR |
| 115 27 |
| Greece |
| Novartis Investigative Site | Pátrai | 265 00 | Greece |
| Novartis Investigative Site | Thessaloniki | 54642 | Greece |
| Novartis Investigative Site | Catania | CT | 95125 | Italy |
| Novartis Investigative Site | Cona | FE | 44100 | Italy |
| Novartis Investigative Site | Genova | GE | 16128 | Italy |
| Novartis Investigative Site | Cagliari | ITA | 09121 | Italy |
| Novartis Investigative Site | Lecce | LE | 73100 | Italy |
| Novartis Investigative Site | Milan | MI | 20122 | Italy |
| Novartis Investigative Site | Palermo | PA | 90127 | Italy |
| Novartis Investigative Site | Palermo | PA | 90146 | Italy |
| Novartis Investigative Site | Verona | VR | 37126 | Italy |
| Novartis Investigative Site | Naples | 80138 | Italy |
| PR - death |
|
| PR - adverse event |
|
| PR - abnormal lab value(s) |
|
| PR- unwilling to comply with procedures |
|
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Number | Participants |
|
| Main underlying disease | Participants with myelodysplastic syndrome (MDS) and transfusion-dependent thalassemia. The very low, low or intermediate risk MDS was to be determined by the Revised International Prognostic Scoring System (IPSS-R) and IPSS-R was to be confirmed by a bone marrow examination within 6 months prior to study entry. MDS with INT risk =MDS with intermediate risk | Number | participants |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Primary | Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest | Posted | Mean | Standard Deviation | 10^12 cells/L | Baseline, 6 and 12 months |
|
|
|
| Primary | Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest | Posted | Mean | Standard Deviation | 10^9 cells/L | Baseline, 6 and 12 months |
|
|
|
| Primary | Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest | Posted | Mean | Standard Deviation | 10^9 cells/L | Baseline, 6 and 12 months |
|
|
|
| Primary | Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest | Posted | Mean | Standard Deviation | umol/L | Baseline, 6 and 12 months |
|
|
|
| Primary | Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest | Posted | Mean | Standard Deviation | mL/min | Baseline, 6 and 12 months |
|
|
|
| Primary | Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest | Posted | Mean | Standard Deviation | U/L | Baseline, 6 and 12 months |
|
|
|
| Primary | Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest | Posted | Mean | Standard Deviation | U/L | Baseline, 6 and 12 months |
|
|
|
| Secondary | Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline. A negative change from baseline is regarded as an improvement in this study | Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest | Posted | Mean | Standard Deviation | ug/l | Baseline, 6 and 12 months |
|
|
|
| Secondary | Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12 | The percentage relative change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point. A negative percentage relative change from baseline is regarded as an improvement in this study | Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest | Posted | Mean | Standard Deviation | percentage of relative change | Baseline, 6 and 12 months |
|
|
|
| 1 |
| 53 |
| 13 |
| 53 |
| 48 |
| 53 |
| Cardiac failure | Cardiac disorders | MedDRA (22.0) | Systematic Assessment |
|
| Goitre | Endocrine disorders | MedDRA (22.0) | Systematic Assessment |
|
| Biliary colic | Hepatobiliary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Cholecystitis | Hepatobiliary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Cholestasis | Hepatobiliary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Hepatic failure | Hepatobiliary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Diverticulitis | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Lower respiratory tract infection | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Lymph gland infection | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Urosepsis | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Femur fracture | Injury, poisoning and procedural complications | MedDRA (22.0) | Systematic Assessment |
|
| Fracture | Injury, poisoning and procedural complications | MedDRA (22.0) | Systematic Assessment |
|
| Lumbar vertebral fracture | Injury, poisoning and procedural complications | MedDRA (22.0) | Systematic Assessment |
|
| Rib fracture | Injury, poisoning and procedural complications | MedDRA (22.0) | Systematic Assessment |
|
| Ulna fracture | Injury, poisoning and procedural complications | MedDRA (22.0) | Systematic Assessment |
|
| Malignant melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (22.0) | Systematic Assessment |
|
| Ovarian adenoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (22.0) | Systematic Assessment |
|
| Papillary thyroid cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (22.0) | Systematic Assessment |
|
| Abortion spontaneous | Pregnancy, puerperium and perinatal conditions | MedDRA (22.0) | Systematic Assessment |
|
| Device failure | Product Issues | MedDRA (22.0) | Systematic Assessment |
|
| Panic attack | Psychiatric disorders | MedDRA (22.0) | Systematic Assessment |
|
| Calculus urinary | Renal and urinary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Hydronephrosis | Renal and urinary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Renal colic | Renal and urinary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Ureterolithiasis | Renal and urinary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA (22.0) | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (22.0) | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA (22.0) | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (22.0) | Systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA (22.0) | Systematic Assessment |
|
| Gastritis | Gastrointestinal disorders | MedDRA (22.0) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (22.0) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (22.0) | Systematic Assessment |
|
| Asthenia | General disorders | MedDRA (22.0) | Systematic Assessment |
|
| Influenza like illness | General disorders | MedDRA (22.0) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (22.0) | Systematic Assessment |
|
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Respiratory tract infection | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Tonsillitis | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA (22.0) | Systematic Assessment |
|
| Blood creatinine increased | Investigations | MedDRA (22.0) | Systematic Assessment |
|
| Urine protein/creatinine ratio increased | Investigations | MedDRA (22.0) | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (22.0) | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (22.0) | Systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (22.0) | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (22.0) | Systematic Assessment |
|
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (22.0) | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (22.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (22.0) | Systematic Assessment |
|
| Dysuria | Renal and urinary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Glycosuria | Renal and urinary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Proteinuria | Renal and urinary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Renal colic | Renal and urinary disorders | MedDRA (22.0) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (22.0) | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (22.0) | Systematic Assessment |
|
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (22.0) | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA (22.0) | 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.
| D000743 | Anemia, Hemolytic |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D001555 |
| Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D014230 | Triazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
|
| Change |
|
|
| Change |
|
|
| Change |
|
|
| Change |
|
|
| Change |
|
|
| Change |
|
|
| Change |
|
|
| Change |
|