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Iron, one of the most common elements in nature and the most abundant transition metal in the body, is readily capable of accepting and donating electrons. This capability makes iron a useful component of various, essential biochemical processes. Despite the essential role of iron, the excess of iron is toxic to the human body. It is critical for the human body to maintain iron balance, since humans have no physiologic mechanism for actively removing iron from the body.
The development of iron overload occurs when iron intake exceeds the body's capacity to safely store the iron in the liver, which is the primary store for iron. Long-term transfusion therapy, a life-giving treatment for patients with intractable chronic anemia is currently the most frequent cause of secondary iron overload.
The mounting evidence regarding the mortality and morbidity due to chronic iron overload in transfusion dependent anaemias has led to the establishment of guidelines that aim the improvement of patient outcomes. Further prospective studies are warranted in order to assess the impact of iron overload in patients with acquired anaemias.
In this study, non-invasive R2- and T2*-MRI techniques were applied to the liver and the heart, respectively, to complement the primary variable (serum ferritin) assessed in patients with various transfusion-dependent anaemias. The main objective of this study was to assess the prevalence and severity of cardiac and liver siderosis in patients with transfusional siderosis. This study was also aim to establish possible correlations between cardiac and liver iron levels with clinical effects in patients with different transfusion-dependent anaemias. Patients were eligible for enrollment irrespective of receiving chelation therapy or not (and irrespective of the chelating agent used).
This study was designed to collect information about a large cohort of patients with anaemias including MDS, aplastic anemia, Diamond-Blackfan, myeloproliferative disorder, as well as haemoglobinopathies (e.g. thalassaemia major, SCD) or other anaemias requiring chronic red blood cell transfusions.
Clinical data was collected retrospectively (if available), unless specified by this protocol (e.g. serum ferritin within less than one month prior to enrollment). All assessments required for this protocol were performed after the patient informed consent is signed. The data was gathered by all study centers and was combined in one central database.
Data was recorded using an electronic case report form (eCRF) at each study site. Adverse events and serious adverse events were recorded for all patients from the date of signed patient informed consent until the MRI tests are performed.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Magnetic Resonance Imaging (MRI) | Other | All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MRI scan | Device | MRI was used to measure both liver and cardiac iron loading (R2 by FerriScan and T2*, respectively). |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Cardiac and Liver Iron Overload. | Hepatic iron overload (liver siderosis) and cardiac iron overload (cardiac siderosis) in patients with transfusional siderosis (Myelodysplastic syndrome (MDS), thalassaemia major, non-transfusion-dependent thalassaemia (NTDT) and other anaemias) were measured using MRI (R2 by FerriScan and T2*, respectively). | 2 months |
| Cardiac Siderosis Severity | Cardiac siderosis severity was measured by MRI (T2*). The severity grade of siderosis was tiered in 3 levels: mild (T2* >= 20ms), moderate (T2* from 10 to 20ms), and severe (T2* <10ms). Mild cardiac siderosis, by the definitions used in this study, were equivalent to not having cardiac siderosis. Values were compared to published thresholds of iron overload to determine severity of transfusion siderosis in the participant population studied. | 2 months |
| Measure | Description | Time Frame |
|---|---|---|
| Comparison of T2* Levels to Evaluate the Severity of Iron Overload Due to Transfusion Therapy in Chelation-naïve and Chelation-treated Participant Subgroups | Iron overload due to transfusion therapy was assessed based on chelation status of each participant (i.e. minimally exposed to chelator treatment and chelation-treated patient subgroups). | 2 months |
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Inclusion Criteria:
Exclusion Criteria:
Any condition that does not allow the MRI test to be performed: 1. Cardiac pacemaker, 2. Ferromagnetic metal implants other than those approved as safe for use in MR scanners (Example: some types of aneurysm clips, shrapnel), 3. Obesity (exceeding the equipment limits), 4. Patients who are claustrophobic to MR Women who are pregnant Unwillingness or being unable to give consent
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Camperdown | New South Wales | 2050 | Australia | ||
| Novartis Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31723837 | Derived | Ho PJ, Hiwase D, Ramakrishna R, Viiala N, Solterbeck A, Traficante R, Zor E, Gervasio OL, High LM, Ross DM, Bowden DK. Cardiac and hepatic siderosis in myelodysplastic syndrome, thalassemia and diverse causes of transfusion-dependent anemia: the TIMES study. Hemasphere. 2019 Jun 4;3(3):e224. doi: 10.1097/HS9.0000000000000224. eCollection 2019 Jun. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Magnetic Resonance Imaging (MRI) | All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Comparison of Liver Iron Concentration (LIC) Levels to Evaluate Iron Overload Due to Transfusion Therapy in Chelation-naïve and Chelation-treated Participant Subgroups | Iron overload due to transfusion therapy was assessed based on chelation status of each participant (i.e. minimally exposed to chelator treatment and chelation-treated patient subgroups). The mean data presented are mean estimates of log transformed data. | 2 months |
| Mean Serum Ferritin According to the Presence or Absence of Retrospective Cardiac Events | Mean serum ferritin according to the presence or absence of cardiac events was assessed for all participant subgroups. | 12 months - retrospective |
| Mean Serum Ferritin According to the Presence or Absence of Retrospective Hepatic Events | Mean serum ferritin according to the presence or absence of hepatic events was assessed for all participant subgroups. | 12 months - retrospective |
| Mean Cardiac T2* According to the Presence or Absence of Retrospective Cardiac Events | Mean cardiac T2* according to the presence or absence of cardiac events was assessed for all participant subgroups. The mean data presented are mean estimates of log transformed data. | 12 months - retrospective |
| Mean LIC According to the Presence or Absence of Retrospective Hepatic Events | Mean LIC according to the presence or absence of hepatic events was assessed for all participant subgroups. | 12 months - retrospective |
| Mean Blood Magnetic Susceptibility (BMS) | Blood samples were collected to assess BMS. The measurement represents absolute magnetic susceptibility at 1 month. Whole blood magnetic susceptibility was calculated by the addition of the dry weight susceptibility and the contribution of the water driven from the sample. | 1 month |
| Percentage of Participants Transfused With Erythrocytes | Transfusion requirement in participants with acquired anaemias with history of receiving chelation therapy was assessed. | 12 months - retrospective |
| Percentage of Participants With Time Since Most Recent Transfuison of <7 Days, 7 to < 14 Days, 14 to < 30 Days, 30 to < 60 Days or >= 60 Days | Transfusion requirement in participants with acquired anaemias with history of receiving chelation therapy was assessed. | 12 months - retrospective |
| Mean Number of Erythrocyte Units Transfused in Last 12 Months | Transfusion requirement in participants with acquired anaemias with history of receiving chelation therapy was assessed. | 12 months - retrospective |
| Mean Quality of Life (QOL) Scores | Quality of life was assessed using the Short Form 36 (SF-36) Health Survey. The SF-36 consists of 8 sub-scales: vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning and mental health. The raw sores of the 8 scales are transformed to a 0 - 100 scale where 0 indicates maximum disability and 100 indicates no disability. There also are two physical and mental health summary measures. Each summary measure is the mean average of the 4 associated sub-scale scores. The range for each summary measure is 0 to 100 where 0 represents maximum disability and 100 represents no disability. | 1 month |
| Percentage of Participants With Low Medium or High Adherence to Iron Chelator Therapy | Adherence of participants was assessed using an adherence questionnaire. Adherence questionnaires were completed only by participants who received chelating agents. Participants answered yes or no to 6 statements such as "Forgot to take pills". Based on the responses to these questions, adherence was classified as low, medium or high. | 1 month |
| Investigator Treatment Decisions Based on MRI Results | Treatment decisions were recorded after the investigator evaluated the MRI results, in order to assess the impact of such diagnostic test on the overall clinical management of participants with iron overload. Investigators answered the following question: "Since the MRI scan, have you changed or are planning to change the management of iron in your subject?". | 2 months |
| Kogarah |
| New South Wales |
| 2217 |
| Australia |
| Novartis Investigative Site | Liverpool | New South Wales | 2170 | Australia |
| Novartis Investigative Site | St Leonards | New South Wales | 2065 | Australia |
| Novartis Investigative Site | Wollongong | New South Wales | 2500 | Australia |
| Novartis Investigative Site | South Brisbane | Queensland | 4101 | Australia |
| Novartis Investigative Site | Woolloongabba | Queensland | 4102 | Australia |
| Novartis Investigative Site | Adelaide | South Australia | 5000 | Australia |
| Novartis Investigative Site | Bedford Park | South Australia | 5042 | Australia |
| Novartis Investigative Site | Hobart | Tasmania | 7000 | Australia |
| Novartis Investigative Site | East Bentleigh | Victoria | 3165 | Australia |
| Novartis Investigative Site | Nedlands | Western Australia | 6009 | Australia |
| Novartis Investigative Site | Perth | Western Australia | 6000 | Australia |
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Magnetic Resonance Imaging (MRI) | All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | 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 | Percentage of Participants With Cardiac and Liver Iron Overload. | Hepatic iron overload (liver siderosis) and cardiac iron overload (cardiac siderosis) in patients with transfusional siderosis (Myelodysplastic syndrome (MDS), thalassaemia major, non-transfusion-dependent thalassaemia (NTDT) and other anaemias) were measured using MRI (R2 by FerriScan and T2*, respectively). | Only participants with valid T2* by MRI and valid liver iron concentration (LIC) by MRI were included for the cardiac siderosis and liver siderosis analyses, respectively. | Posted | Number | Percentage of participants | 2 months |
|
|
| ||||||||||||||||||||||||||||||||||||||
| Primary | Cardiac Siderosis Severity | Cardiac siderosis severity was measured by MRI (T2*). The severity grade of siderosis was tiered in 3 levels: mild (T2* >= 20ms), moderate (T2* from 10 to 20ms), and severe (T2* <10ms). Mild cardiac siderosis, by the definitions used in this study, were equivalent to not having cardiac siderosis. Values were compared to published thresholds of iron overload to determine severity of transfusion siderosis in the participant population studied. | Only participants with valid T2* by MRI were included in the analysis. | Posted | Number | Percentage of participants | 2 months |
| ||||||||||||||||||||||||||||||||||||||||
| Secondary | Comparison of T2* Levels to Evaluate the Severity of Iron Overload Due to Transfusion Therapy in Chelation-naïve and Chelation-treated Participant Subgroups | Iron overload due to transfusion therapy was assessed based on chelation status of each participant (i.e. minimally exposed to chelator treatment and chelation-treated patient subgroups). | Only participants with valid T2* by MRI were included in the analysis. | Posted | Least Squares Mean | 95% Confidence Interval | ms | 2 months |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Comparison of Liver Iron Concentration (LIC) Levels to Evaluate Iron Overload Due to Transfusion Therapy in Chelation-naïve and Chelation-treated Participant Subgroups | Iron overload due to transfusion therapy was assessed based on chelation status of each participant (i.e. minimally exposed to chelator treatment and chelation-treated patient subgroups). The mean data presented are mean estimates of log transformed data. | Only participants with valid LIC by MRI were included in the analysis. | Posted | Mean | 95% Confidence Interval | mg Fe/g | 2 months |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Serum Ferritin According to the Presence or Absence of Retrospective Cardiac Events | Mean serum ferritin according to the presence or absence of cardiac events was assessed for all participant subgroups. | Participants with serum ferritin values and previous cardiac events data were included in the analysis. | Posted | Mean | Standard Deviation | ng/mL | 12 months - retrospective |
|
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Serum Ferritin According to the Presence or Absence of Retrospective Hepatic Events | Mean serum ferritin according to the presence or absence of hepatic events was assessed for all participant subgroups. | Participants with serum ferritin values and previous hepatic events data were included in the analysis. | Posted | Mean | Standard Deviation | ng/mL | 12 months - retrospective |
|
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Cardiac T2* According to the Presence or Absence of Retrospective Cardiac Events | Mean cardiac T2* according to the presence or absence of cardiac events was assessed for all participant subgroups. The mean data presented are mean estimates of log transformed data. | Participants with valid T2* by MRI results were included in the analysis. | Posted | Mean | Standard Deviation | log10 (ms) | 12 months - retrospective |
|
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Mean LIC According to the Presence or Absence of Retrospective Hepatic Events | Mean LIC according to the presence or absence of hepatic events was assessed for all participant subgroups. | Participants with LIC by MRI were included in the analysis. | Posted | Mean | Standard Deviation | mg Fe/g | 12 months - retrospective |
|
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Blood Magnetic Susceptibility (BMS) | Blood samples were collected to assess BMS. The measurement represents absolute magnetic susceptibility at 1 month. Whole blood magnetic susceptibility was calculated by the addition of the dry weight susceptibility and the contribution of the water driven from the sample. | Participants with BMS values were analyzed. | Posted | Mean | Standard Deviation | emu/g wet wt/Oe | 1 month |
|
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants Transfused With Erythrocytes | Transfusion requirement in participants with acquired anaemias with history of receiving chelation therapy was assessed. | Participants with a erythrocyte transfusion history were included in the analysis. | Posted | Number | Percentage of participants | 12 months - retrospective |
| ||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Time Since Most Recent Transfuison of <7 Days, 7 to < 14 Days, 14 to < 30 Days, 30 to < 60 Days or >= 60 Days | Transfusion requirement in participants with acquired anaemias with history of receiving chelation therapy was assessed. | Participants with data on time since their most recent transfusion were included in the analysis. | Posted | Number | percentage of participants | 12 months - retrospective |
| ||||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Number of Erythrocyte Units Transfused in Last 12 Months | Transfusion requirement in participants with acquired anaemias with history of receiving chelation therapy was assessed. | Participants with 'number of units transfused' data were included in the analysis. | Posted | Mean | Standard Deviation | number of units transfused | 12 months - retrospective |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Quality of Life (QOL) Scores | Quality of life was assessed using the Short Form 36 (SF-36) Health Survey. The SF-36 consists of 8 sub-scales: vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning and mental health. The raw sores of the 8 scales are transformed to a 0 - 100 scale where 0 indicates maximum disability and 100 indicates no disability. There also are two physical and mental health summary measures. Each summary measure is the mean average of the 4 associated sub-scale scores. The range for each summary measure is 0 to 100 where 0 represents maximum disability and 100 represents no disability. | Only participants with data for each subscale were included in the analysis for that subscale. | Posted | Mean | Standard Deviation | units on a scale | 1 month |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Low Medium or High Adherence to Iron Chelator Therapy | Adherence of participants was assessed using an adherence questionnaire. Adherence questionnaires were completed only by participants who received chelating agents. Participants answered yes or no to 6 statements such as "Forgot to take pills". Based on the responses to these questions, adherence was classified as low, medium or high. | Participants who were on iron chelator therapy at screening, and had answered at least one question on the questionnaire and had sufficient information to score the questionnaire, were included in the analysis. | Posted | Number | Percentage of participants | 1 month |
| ||||||||||||||||||||||||||||||||||||||||
| Secondary | Investigator Treatment Decisions Based on MRI Results | Treatment decisions were recorded after the investigator evaluated the MRI results, in order to assess the impact of such diagnostic test on the overall clinical management of participants with iron overload. Investigators answered the following question: "Since the MRI scan, have you changed or are planning to change the management of iron in your subject?". | Participants, for whom treatment decision questionnaire results were provided and for whom MRI results were available, were included in the analysis. | Posted | Number | Percentage of participants | 2 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 | Thalassaemia Major | Subset of overall participants with thalassemia major | 2 | 81 | 7 | 81 | ||
| EG001 | Myelodysplastic Syndrome (MDS) | Subset of overall participants with MDS | 8 | 74 | 9 | 74 | ||
| EG002 | Other Anaemia | Subset of overall participants with other types of anaemias | 2 | 68 | 3 | 68 | ||
| EG003 | Non-transfusion-dependent Anaemia (NTDT) | Subset of overall participants with NTDT | 0 | 20 | 0 | 20 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Eye swelling | Eye disorders | MedDRA | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA | Systematic Assessment |
| |
| Medical device site erosion | General disorders | MedDRA | Systematic Assessment |
| |
| Medical device site pain | General disorders | MedDRA | Systematic Assessment |
| |
| Medical device site swelling | General disorders | MedDRA | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA | Systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Klebsiella infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA | Systematic Assessment |
| |
| Electrocardiogram QT prolonged | Investigations | MedDRA | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Myelodysplastic syndrome | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Hypoacusis | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Gastrointestinal disorder | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Proctalgia | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA | Systematic Assessment |
| |
| Injection site reaction | General disorders | MedDRA | Systematic Assessment |
| |
| Oedema | General disorders | MedDRA | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA | Systematic Assessment |
| |
| Pain | General disorders | MedDRA | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Heart rate increased | Investigations | MedDRA | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Gout | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Sinus headache | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Sleep disorder | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA | 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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis | 862-778-8300 |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| D019190 | Iron Overload |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| D008279 | Magnetic Resonance Imaging |
| ID | Term |
|---|---|
| D014054 | Tomography |
| D003952 | Diagnostic Imaging |
| D019937 | Diagnostic Techniques and Procedures |
| D003933 | Diagnosis |
Not provided
Not provided
| Liver |
|
Subset of overall participants with NTDT |
| OG004 | Other Anaemias | Subset of overall participants with other types of anaemia |
|
|
| OG004 | Other Anaemias | Subset of overall participants with other types of anaemia |
|
|
Subset of overall participants with NTDT
| OG004 | Other Anaemias | Subset of overall participants with other types of anaemia |
|
|
|
|
|
|
|
Subset of overall participants with other types of anaemia |
|
|
| OG004 |
| Other Anaemias |
Subset of overall participants with other types of anaemia |
|
|
| Other Anaemias |
Subset of overall participants with other types of anaemia |
|
|
| OG003 | Non-transfusion-dependent Anaemia (NTDT) | Subset of overall participants with NTDT |
| OG004 | Other Anaemias | Subset of overall participants with other types of anaemia |
|
|
Subset of overall participants with NTDT |
| OG004 | Other Anaemias | Subset of overall participants with other types of anaemia |
|
|
|