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| ID | Type | Description | Link |
|---|---|---|---|
| EudraCT no. 2006-002102-57 | Registry Identifier | EudraCT |
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Brief Summary: This study was designed to explore a safe dose and characterize the preliminary safety and efficacy of ICL670 in adult patients with previously documented history of homozygous C282Y.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ICL670 (Deferasirox) | Experimental | Three dose cohorts: 5 mg/kg/day, 10 mg/kg/day, 15 mg/kg/day |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deferasirox (ICL670) | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population) | Mean absolute change in serum ferritin from baseline to the end of the extension study. | 0 to 48 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population) | A blood sample was collected just prior to administration of the next dose of Deferasirox (pre-dose trough level) or approximately 24 hours after the previous dose at weeks 4, 8, 12, 16, 20 and 24. The mean trough concentration at each time point was calculated. | 4, 8, 12, 16, 20, and 24 weeks |
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Inclusion Criteria:
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply.
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals, M.D. | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UC Irvine/Long Beach | Long Beach | California | 90822 | United States | ||
| University of Connecticut Health Center |
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| Label | URL |
|---|---|
| Related Info | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Deferasirox (ICL670) 5 mg/kg/Day | Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. |
| FG001 | Deferasirox (ICL670) 10 mg/kg/Day |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Core Study |
|
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| Farmington |
| Connecticut |
| 06030 |
| United States |
| Henry Ford Health System | Detroit | Michigan | 48202 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| St. Louis University | St Louis | Missouri | 63110 | United States |
| Rochester General Hospital | Rochester | New York | 14625 | United States |
| Carolinas Medical Center | Charlotte | North Carolina | 28203 | United States |
| Ohio State University Medical Center | Columbus | Ohio | 43210 | United States |
| Three Medical Park | Columbia | South Carolina | 29203 | United States |
| Novartis Investigative Site | Brisbane | Australia |
| Novartis Investigative Site | London | Ontario | Canada |
| Novartis Investigative Site | Rennes | France |
| Novartis Investigative Site | Chemnitz | Germany |
| Novartis Investigative Site | Heidelberg | Germany |
| Novartis Investigative Site | Magdeburg | Germany |
| Novartis Investigative Site | Oberhausen | Germany |
| Novartis Investigative Site | Modena | Italy |
| Novartis Investigative Site | Monza | Italy |
Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. |
| FG002 | Deferasirox (ICL670) 15 mg/kg/Day | Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. |
| COMPLETED |
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| NOT COMPLETED |
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| Extension Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Deferasirox (ICL670) 5 mg/kg/Day | Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. |
| BG001 | Deferasirox (ICL670) 10 mg/kg/Day | Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. |
| BG002 | Deferasirox (ICL670) 15 mg/kg/Day | Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age Continuous | The n values in the Extension Study Category are the number of participants in each Arm/Group. The last n value= 26 is the Total number of participants. | Mean | Standard Deviation | years |
| ||||||||||||||
| Sex/Gender, Customized | Number | participants |
| ||||||||||||||||
| Sex/Gender, Customized | Number | participants |
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| 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 | Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population) | Mean absolute change in serum ferritin from baseline to the end of the extension study. | Extension Per-protocol population including all participants of the per protocol population who also were part of the extension safety population. | Posted | Mean | Standard Deviation | µg/L | 0 to 48 weeks |
|
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| |||||||||||||||||||||||||||||||
| Secondary | Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population) | A blood sample was collected just prior to administration of the next dose of Deferasirox (pre-dose trough level) or approximately 24 hours after the previous dose at weeks 4, 8, 12, 16, 20 and 24. The mean trough concentration at each time point was calculated. | Per-protocol population included all participants of the safety population; who received at least one dose of study drug within the core study and had at least one safety assessment, and who did not have any major protocol deviation. n in each of the Categories is the number of participants in each arm/group who had data for that time point. | Posted | Mean | Standard Deviation | µmol/L | 4, 8, 12, 16, 20, and 24 weeks |
|
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Safety Population includes all participants who received at least one dose of study drug and had at least one safety assessment.
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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 (ICL670) 5 mg/kg/day_Core Study | Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. | 0 | 11 | 10 | 11 | ||
| EG001 | Deferasirox (ICL670) 10 mg/kg/day_Core Study | Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. | 0 | 15 | 14 | 15 | ||
| EG002 | Deferasirox (ICL670) 15 mg/kg/day_Core Study | Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. | 0 | 23 | 23 | 23 | ||
| EG003 | Deferasirox (ICL670) 5 mg/kg/day_Extension Study | Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. | 1 | 9 | 9 | 9 | ||
| EG004 | Deferasirox (ICL670) 10 mg/kg/day_Extension Study | Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. | 0 | 6 | 5 | 6 | ||
| EG005 | Deferasirox (ICL670) 15 mg/kg/day_Extension Study | Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. | 0 | 11 | 11 | 11 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Prostate cancer recurrent | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Arrhythmia | Cardiac disorders | MedDRA | Systematic Assessment |
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| Atrioventricular block first degree | Cardiac disorders | MedDRA | Systematic Assessment |
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| Hydrocele | Congenital, familial and genetic disorders | MedDRA | Systematic Assessment |
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| Ear pain | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
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| Hypoacusis | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
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| Mixed deafness | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
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| Neurosensory hypoacusis | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
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| Tinnitus | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
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| Vertigo | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
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| Blindness day | Eye disorders | MedDRA | Systematic Assessment |
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| Cataract | Eye disorders | MedDRA | Systematic Assessment |
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| Corneal opacity | Eye disorders | MedDRA | Systematic Assessment |
| |
| Eye pain | Eye disorders | MedDRA | Systematic Assessment |
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| Maculopathy | Eye disorders | MedDRA | Systematic Assessment |
| |
| Presbyopia | Eye disorders | MedDRA | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA | Systematic Assessment |
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| Visual acuity reduced | Eye disorders | MedDRA | Systematic Assessment |
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| Visual impairment | Eye disorders | MedDRA | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Dyspepsia | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Flatulence | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Frequent bowel movements | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Gingival disorder | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Gingival pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Glossodynia | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Retching | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Asthenia | General disorders | MedDRA | Systematic Assessment |
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| Chest discomfort | General disorders | MedDRA | Systematic Assessment |
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| Fatigue | General disorders | MedDRA | Systematic Assessment |
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| Influenza like illness | General disorders | MedDRA | Systematic Assessment |
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| Non-cardiac chest pain | General disorders | MedDRA | Systematic Assessment |
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| Oedema peripheral | General disorders | MedDRA | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA | Systematic Assessment |
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| Hepatic function abnormal | Hepatobiliary disorders | MedDRA | Systematic Assessment |
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| Drug hypersensitivity | Immune system disorders | MedDRA | Systematic Assessment |
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| Seasonal allergy | Immune system disorders | MedDRA | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA | Systematic Assessment |
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| Lower respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Oral herpes | Infections and infestations | MedDRA | Systematic Assessment |
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| Pharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
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| Vulvovaginal mycotic infection | Infections and infestations | MedDRA | Systematic Assessment |
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| Eye injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA | Systematic Assessment |
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| Aspartate aminotransferase increased | Investigations | MedDRA | Systematic Assessment |
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| Blood alkaline phosphatase increased | Investigations | MedDRA | Systematic Assessment |
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| Blood cholesterol increased | Investigations | MedDRA | Systematic Assessment |
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| Blood creatine increased | Investigations | MedDRA | Systematic Assessment |
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| Blood creatinine increased | Investigations | MedDRA | Systematic Assessment |
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| C-reactive protein increased | Investigations | MedDRA | Systematic Assessment |
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| Creatinine renal clearance decreased | Investigations | MedDRA | Systematic Assessment |
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| Creatinine renal clearance increased | Investigations | MedDRA | Systematic Assessment |
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| Gamma-glutamyltransferase increased | Investigations | MedDRA | Systematic Assessment |
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| Transaminases increased | Investigations | MedDRA | Systematic Assessment |
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| White blood cell count increased | Investigations | MedDRA | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Musculoskeletal discomfort | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Tendonitis | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Prostate cancer recurrent | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
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| Hypoaesthesia | Nervous system disorders | MedDRA | Systematic Assessment |
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| Memory impairment | Nervous system disorders | MedDRA | Systematic Assessment |
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| Migraine | Nervous system disorders | MedDRA | Systematic Assessment |
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| Neuralgia | Nervous system disorders | MedDRA | Systematic Assessment |
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| Syncope vasovagal | Nervous system disorders | MedDRA | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA | Systematic Assessment |
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| Insomnia | Psychiatric disorders | MedDRA | Systematic Assessment |
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| Calculus urinary | Renal and urinary disorders | MedDRA | Systematic Assessment |
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| Renal impairment | Renal and urinary disorders | MedDRA | Systematic Assessment |
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| Benign prostatic hyperplasia | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
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| Erectile dysfunction | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Laryngeal inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Actinic keratosis | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Ingrowing nail | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Precancerous skin lesion | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Rash generalised | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Seborrhoeic dermatitis | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Urticaria | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Hot flush | Vascular disorders | MedDRA | Systematic Assessment |
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| Hypertension | Vascular disorders | MedDRA | Systematic Assessment |
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| Hypotension | Vascular disorders | MedDRA | Systematic Assessment |
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| Orthostatic hypotension | Vascular disorders | MedDRA | Systematic Assessment |
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| Peripheral coldness | Vascular disorders | MedDRA | Systematic Assessment |
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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 (i.e., data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-8300 |
| ID | Term |
|---|---|
| D019190 | Iron Overload |
| D006432 | Hemochromatosis |
| ID | Term |
|---|---|
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D008664 | Metal Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D000077588 | Deferasirox |
| ID | Term |
|---|---|
| D001565 | Benzoates |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D014230 | Triazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
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| Extension Study (n=9,6,11,26) |
|
| Male_Core Study |
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| Male_Extension Study |
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| Core Participants not enrolled in the Extension |
|
Deferasirox (ICL670) was provided as 125 mg, 250 mg, and 500 mg tablets. Dosage was based on the participant's body weight. ICL670 was administered orally, once a day, 30 minutes prior to breakfast. |
|
|