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This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant period in patients with beta-thalassemia major.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ICL670 | Experimental | Oral dose of ICL670 at 10 mg/kg daily |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ICL670 | Drug | Oral dose of ICL670 at 10 mg/kg daily |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability | To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after hematopoietic stem cell transplantation (HSCT) in patients with beta-thalassemia major in 12 months period | 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Serum Ferritin Level. | Blood samples were collected and serum levels were assessed at study baseline (BL) and at 12 months. | Baseline, 12 Months |
| Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination) |
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Inclusion Criteria
A. Serum ferritin >1000 μg/L or B. cardiac MRI <20 ms or C. liver iron concentration ≥ 5 mg/g dry weight measured by R2* MRI
Exclusion Criteria
Patients who had any contraindication for treatment with deferasirox according to the prescribing information
•Patients who depended on transfusion
Patients with clinical symptoms of cardiac dysfunction (shortness of breath at rest or exertion, orthopnea, exercise intolerance, lower extremity edema, arrhythmias)
Patients who were experiencing severe complication of HSCT e.g. acute Graft-versus host disease (GVHD)
Significant proteinuria / Increase in serum creatinine
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Ankara | Turkey | 06100 | Turkey (Türkiye) | ||
| Novartis Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 29155313 | Derived | Yesilipek MA, Karasu G, Kaya Z, Kuskonmaz BB, Uygun V, Dag I, Ozudogru O, Ertem M. A Phase II, Multicenter, Single-Arm Study to Evaluate the Safety and Efficacy of Deferasirox after Hematopoietic Stem Cell Transplantation in Children with beta-Thalassemia Major. Biol Blood Marrow Transplant. 2018 Mar;24(3):613-618. doi: 10.1016/j.bbmt.2017.11.006. Epub 2017 Nov 16. |
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| ID | Title | Description |
|---|---|---|
| FG000 | ICL670 | Oral dose of ICL670 at 10 mg/kg daily |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | ICL670 | Oral dose of ICL670 at 10 mg/kg daily |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability | To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after hematopoietic stem cell transplantation (HSCT) in patients with beta-thalassemia major in 12 months period | The Safety Set (SS) includes all included patients who were included in the study. All statistical analyses of safety and tolerability will be done in the SS. | Posted | Number | Participants | 12 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 | ICL670 | Oral dose of ICL670 at 10 mg/kg daily |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Neutrophil count decreased | Investigations | MedDRA 18.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | MedDRA 18.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis | 862-778-8300 |
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| ID | Term |
|---|---|
| D017086 | beta-Thalassemia |
| D019190 | Iron Overload |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
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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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Liver Iron Concentration (LIC) values between 3 and 7 mg Fe / g dry weight are indicative of mild iron deposition, while values between 7 and 15 mg Fe / g dry weight are indicative of moderate iron deposition which have been associated with liver disease. Values >15 mg Fe/g dry weight are indicative of severe iron deposition which is associated with progressive liver fibrosis, increased morbidity and mortality |
| Baseline, 12 month |
| The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L | Serum Ferritin values between 1000-2500 μg/L are indicative of mild to moderate iron overload while values >2500 μg/L are indicative of severe iron overload and levels constantly higher than 2500 μg/L has been shown to to increase the risk of cardiac complications and endocrine disease. Maintaining levels <1000 μg/L is associated with increased survival and less morbidity. | Week 28 and Week 52 |
| Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination) | Cardiac MRI values between 10 to 20 milliseconds (ms) are indicative of moderate cardiac iron deposition associated with declining left ventricular ejection fraction and arrhythmias while values <10 ms are indicative of deposition sufficient to risk cardiac decompensation and associated with overt heart failure and mortality. | Baseline, 12 month |
| Ankara |
| Turkey |
| 06500 |
| Turkey (Türkiye) |
| Novartis Investigative Site | Izmir | Turkey | 35040 | Turkey (Türkiye) |
| Novartis Investigative Site | Antalya | 07000 | Turkey (Türkiye) |
| Novartis Investigative Site | Antalya | 07070 | Turkey (Türkiye) |
| Novartis Investigative Site | Istanbul | Turkey (Türkiye) |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Units | Counts |
|---|
| Participants |
|
|
| Secondary | Change in Serum Ferritin Level. | Blood samples were collected and serum levels were assessed at study baseline (BL) and at 12 months. | The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose. | Posted | Mean | Standard Deviation | ng/mL | Baseline, 12 Months |
|
|
|
| Secondary | Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination) | Liver Iron Concentration (LIC) values between 3 and 7 mg Fe / g dry weight are indicative of mild iron deposition, while values between 7 and 15 mg Fe / g dry weight are indicative of moderate iron deposition which have been associated with liver disease. Values >15 mg Fe/g dry weight are indicative of severe iron deposition which is associated with progressive liver fibrosis, increased morbidity and mortality | The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose. | Posted | Mean | Standard Deviation | mg Fe/g dry liver weight | Baseline, 12 month |
|
|
|
| Secondary | The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L | Serum Ferritin values between 1000-2500 μg/L are indicative of mild to moderate iron overload while values >2500 μg/L are indicative of severe iron overload and levels constantly higher than 2500 μg/L has been shown to to increase the risk of cardiac complications and endocrine disease. Maintaining levels <1000 μg/L is associated with increased survival and less morbidity. | The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose. | Posted | Number | Percentage of Patients | Week 28 and Week 52 |
|
|
|
| Secondary | Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination) | Cardiac MRI values between 10 to 20 milliseconds (ms) are indicative of moderate cardiac iron deposition associated with declining left ventricular ejection fraction and arrhythmias while values <10 ms are indicative of deposition sufficient to risk cardiac decompensation and associated with overt heart failure and mortality. | The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose. | Posted | Mean | Standard Deviation | ms | Baseline, 12 month |
|
|
|
| 3 |
| 27 |
| 23 |
| 27 |
| Alanine aminotransferase increased | Investigations | MedDRA 18.0 | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA 18.0 | Systematic Assessment |
|
| Influenza like illness | General disorders | medDRA 18.0 | Systematic Assessment |
|
| Office visit | Surgical and medical procedures | MedDRA 18.0 | Systematic Assessment |
|
| Hepatitis B | Hepatobiliary disorders | MedDRA 18.0 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 18.2 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 18.0 | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA 18.0 | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
|
| Influenza | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 18.0 | Systematic Assessment |
|
| White blood cell count decreased | Investigations | MedDRA 18.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Systematic Assessment |
|
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 18.0 | Systematic Assessment |
|
| Haemophilus infection | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
|
| Herpes zoster | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
|
| Hypersensitivity | Immune system disorders | MedDRA 18.0 | Systematic Assessment |
|
| Infection | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
|
| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA 18.0 | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA 18.0 | Systematic Assessment |
|
| Influenza like illness | General disorders | MedDRA 18.0 | 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 (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
| D006402 |
| Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| 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 |