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| ID | Type | Description | Link |
|---|---|---|---|
| 2009-012418-38 | EudraCT Number |
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This was a randomized, double-blind trial to evaluate deferasirox vs placebo in patients with myelodysplastic syndromes (low/int-1 risk) and transfusional iron overload .The trial was conducted in 17 countries, started in 2010 and ended in 2018.
This randomized, double blind trial to evaluate deferasirox vs placebo in patients with myelodysplastic syndromes (low/int-1 risk) and transfusional iron overload consisted of four periods, a screening period, a treatment period, a post treatment follow-up period and a survival period. The trial recruitment period lasted until December 2014 and the trial continued for three years from the date the last patient enrolled until February 2018 (last patient last visit date).
Screening period:
The screening period lasting up to 35 days with two screening visits, at least 14 days apart, used to assess patient eligibility. Eligible patients with low or int-1 risk myelodysplastic syndromes (MDS) with transfusional iron overload were randomized in a 2:1 ratio to deferasirox or placebo respectively. Randomization was also stratified using the International prognostic scoring system of low or int-1 MDS and by geographical region (Asian vs non-Asian countries) since the Asian population has been reported to have a longer survival.
The following concomitant medications could be permitted for use while the patient was on study, and information outlining start date(s) and end date(s) of each medication taken were to be recorded on the appropriate eCRF: Erythropoietin (growth factor), G-CSF (growth factor), GM-CSF growth factor), Azacitidine, Thalidomide, Arsenic trioxide, Lenalidomide, Decitabine, Cyclosporine A, Vitamin C supplements (≤ 200 mg/day)
Treatment period:
The dosing schedule was 10 mg/kg/day (once daily) for the first 2 weeks, followed by 20 mg/kg/day (once daily). After 3 months of treatment at the dose of 20mg/kg/day, the dose could be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on the serum ferritin response. Placebo matching to each strength of the active deferasirox was utilized to maintain the double-blind trial design.
During the treatment period patients returned to the investigational site every four weeks for routine procedures and to monitor safety, efficacy and compliance to treatment.
An external Data Monitoring Committee (DMC) monitored patient safety and trial conduct and received a blinded summary of serious adverse events.
All suspected endpoint events were reviewed and adjudicated by the Endpoint Adjudication Committee (EAC) to ensure that all events that were reported were judged uniformly using the same criteria. The first confirmed suspected endpoint event for a patient was counted for the trial's composite primary endpoint, "event free survival". The composite primary endpoint, "event free survival," was defined for a patient as the date randomized to trial treatment to the date of the first documented non-fatal event, related to cardiac and liver function, transformation to AML, or death due to any cause.
When a patient had a non-fatal event, related to cardiac and liver function, and transformation to AML, the trial treatment (deferasirox or placebo) was discontinued. After trial treatment was discontinued, a 28 days post treatment safety assessment for AEs and SAEs was completed. Any patient who died during the treatment or 28 day post treatment safety assessment is represented in the all-cause mortality table in the safety section of this result. After trial treatment was discontinued for a patient, their treatment was un-blinded. Subsequent iron chelation treatment was subject to the patient's and investigator's decision. Patients continued to be followed during the post-treatment evaluation or survival follow up period, depending on their choice.
For patients who did not meet a non-fatal event, study treatment was continued as long as the patient and the treating physician felt it was in the best interest for the patient or until the trial terminated/completed. There was no un-blinding of the trial treatment for patients who terminated trial treatment without meeting a non-fatal event. Patients continued to be followed during the post-treatment evaluation or survival follow up period, depending on their choice.
A patient who discontinued study treatment without meeting a non-fatal component of the composite primary endpoint continued to be evaluated every 3 months. Once a patient stopped study evaluations they were followed for at least every 6 months for overall survival and any iron chelation therapies they are receiving up to the end of study.
Post-treatment evaluation period:
For patients who had a non-fatal event: After treatment termination, all patients were followed for safety (28 days) and then evaluated with visits every three months if they agreed to move into the post treatment evaluation phase.
For patients who did not meet a non-fatal event: After termination of study treatment, if a patient and investigator chose the post-treatment evaluation period, the patient was followed for safety and endpoints at visits occurring every three months.
Survival Follow Up period:
Subsequent to the post treatment evaluation period, or at the end of treatment period, if a patient and treating physician decided that the patient would not participate in the post treatment evaluation period, the patient was followed every 6 months for overall survival and iron chelation therapies.
The end of the study was defined as three years from the date the last patient was enrolled (last patient first visit).
The sample size of 210 patients did not provide sufficient power for testing statistical hypotheses. The statistical analysis was revised accordingly to concentrate on evaluating the treatment effect of deferasirox relative to placebo, and the study phase designation was changed from Phase lll to Phase II. Amendment 4 of the study adjusted the sample size, statistical analysis, and duration of the study and added two secondary endpoints: Hematologic improvement (HI) in terms of erythroid response and Frequency and rate of infections requiring intravenous (IV) antimicrobials. Upon approval of the amendment, patients signed a new consent form and continued the appropriate visit schedule.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Deferasirox | Experimental | 10 mg/kg/day (once daily) for the first 2 weeks of treatment, followed by 20 mg/kg/day (once daily) from Week 2 to End of Treatment. After 3 months of treatment at 20 mg/kg/day, the dose was allowed to be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on serum ferritin responses. When a target serum ferritin level was reached (usually between 500 and 1000 µg/L), the dose could be reduced by 50% to maintain the serum ferritin within the target range. |
|
| Placebo | Placebo Comparator | 10 mg/kg/day (once daily) for the first 2 weeks of treatment, followed by 20 mg/kg/day (once daily) from Week 2 to End of Treatment. After 3 months of treatment at 20 mg/kg/day, the dose was allowed to be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on serum ferritin responses. When a target serum ferritin level was reached (usually between 500 and 1000 µg/L), the dose could be reduced by 50% to maintain the serum ferritin within the target range. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deferasirox | Drug | Deferasirox provided as 125 mg, 250 mg, and 500 mg dispersible tablets for oral use |
|
| Measure | Description | Time Frame |
|---|---|---|
| Event-free Survival | Event-free survival was defined as the time from the date of randomization to the date of the first documented non-fatal event (worsening cardiac function, hospitalization for congestive heart failure, liver function impairment, liver cirrhosis, transformation to AML, as defined in the protocol), or death, whichever occurred first. Participants who did not experience a non-fatal event as of the time of data cut-off (end of study), as well as participants who did not experience a non-fatal event and stopped study participation before the data cut-off, were censored as specified in the protocol. | Day 1 to end of treatment period, approx. 7 years |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Hematologic Improvement (HI) in Terms of Erythroid Response | HI in terms of erythroid responses was assessed based on International Working Group (IWG) criteria, with improvement defined as follows:
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Pacific Cancer Medical Center, Inc. PAC Center | Anaheim | California | 92801 | United States | ||
| Rocky Mountain Cancer Centers RMCC |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32203980 | Derived | Angelucci E, Li J, Greenberg P, Wu D, Hou M, Montano Figueroa EH, Rodriguez MG, Dong X, Ghosh J, Izquierdo M, Garcia-Manero G; TELESTO Study Investigators. Iron Chelation in Transfusion-Dependent Patients With Low- to Intermediate-1-Risk Myelodysplastic Syndromes: A Randomized Trial. Ann Intern Med. 2020 Apr 21;172(8):513-522. doi: 10.7326/M19-0916. Epub 2020 Mar 24. |
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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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The planned sample size of 210 patients randomized in a ratio of 2:1 in favor of deferasirox was based on the feasibility of enrolling the patients and consultations with the Health Authorities.
1 patient rand. to deferasirox arm did not receive study drug & was excl. from the Safety set. Terminated by Sponsor: Patients who had completed at least 3 years of on-treatment period including 28-day follow-up & were still on trial when end-of-study definition (3 years after LPFV) was reached & therefore, the study was terminated as per protocol
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| ID | Title | Description |
|---|---|---|
| FG000 | Deferasirox | 10 mg/kg/day (once daily) for the first 2 weeks of treatment, followed by 20 mg/kg/day (once daily) from Week 2 to End of Treatment. After 3 months of treatment at 20 mg/kg/day, the dose was allowed to be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on serum ferritin responses. When a target serum ferritin level was reached (usually between 500 and 1000 µg/L), the dose could be reduced by 50% to maintain the serum ferritin within the target range |
| Title | Milestones | Reasons Not Completed | ||||
|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 19, 2018 | Feb 25, 2019 |
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Patients, investigator staff, persons performing the assessments, and data analysts remained blind to the identity of the study treatment from the time of randomization until database lock.
| Placebo | Drug | Inactive ingredients used as a placebo comparator, provided as 125 mg, 250 mg, and 500 mg dispersible tablets for oral use |
|
| Day 1 to end of treatment period, approx. 7 years |
| Overall Survival | Overall survival was calculated as the date of death (irrespective of cause) minus date of randomization plus 1. | Day 1 to end of treatment period, approx. 7.4 years |
| Percentage of Participants With Newly Occurring Hypothyroidism Compared to Baseline | As assessed by annual measurement of Thyroid Stimulating Hormone (TSH) and free T4. Hypothyroidism was defined as follows and is inclusive of:
| Day 1 to end of treatment period, approx. 7 years |
| Percentage of Participants With Worsening Glucose Metabolism Compared to Baseline | As assessed by an annual glucose tolerance test (OGTT). Worsening glucose metabolism was defined as an increase in glucose metabolism category (normal, impaired glucose metabolism, diabetes mellitus) based on the American Diabetes Association criteria (American Diabetes Association 2009) compared to the baseline result. The last available lab assessment date was used as the cut-off date for the analysis. | Day 1 to end of treatment period, approx. 7 years |
| Time to Disease Progression | Disease progression was defined as follows:
Disease progression was calculated as follows: Date of diagnosis of MDS progression or date of first diagnosis of AML, minus date of randomization plus 1. Participants who neither experienced MDS progression nor progression to AML were censored at the last contact date. | Day 1 to end of treatment period, approx. 7 years |
| Time to First Occurrence of Serum Ferritin Level >2 Times the Baseline Value at Two Consecutive Assessments (at Least Two Weeks Apart) | Assessed by blood draw and calculated as follows: Date of first occurrence of serum ferritin >2 times the baseline value at two consecutive assessments (at least two weeks apart), minus date of randomization plus 1. Participants who did not experience such an increase were censored at the last date when serum ferritin was available. | Day 1 to end of treatment period, approx. 7 years |
| Time to at Least a 10% Increase From Baseline in Left Ventricular End-diastolic Internal (LVIDD) at Two Consecutive Assessments at Least Two Weeks Apart | Assessed by echocardiography and calculated as follows: Date of echocardiography assessment where a minimum of 10% increase of LVIDD first occurred, minus date of randomization plus 1. Participants who did not experience such an increase were censored at the last date when LVIDD was available. | Day 1 to end of treatment period, approx. 7 years |
| Time to at Least a 10% Increase From Baseline in Left Ventricular Internal Systolic Diameter (LVISD) at Two Consecutive Assessments at Least Two Weeks Apart | Assessed by echocardiography and calculated as follows: Date of echocardiography assessment where a minimum of 10% increase of LVISD first occurred, minus date of randomization plus 1. Participants who did not experience such an increase were censored at the last date when LVISD was available. | Day 1 to end of treatment period, approx. 7 years |
| Total Number of Infections Requiring Intravenous Antimicrobials | The total number of infections were counted and summarized per treatment group. For this number, one participant can contribute more than one infection event. Infections were determined from the reported AEs with system organ class "Infections and infestations" and action taken "Concomitant medication taken." Antimicrobial therapy was determined from the reported concomitant medications for participants who had an infection AE. The route of administration needed to be specified as "intravenous (i.v.)". End of treatment period was defined as the treatment period plus 28 days. | Day 1 to end of treatment period, approx. 7 years |
| Percentage of Participants With Major Gastrointestinal Bleeding | Major gastrointestinal bleeding was defined as an AE that could include one of the following MedDRA preferred terms: gastric hemorrhage, gastrointestinal hemorrhage, small intestinal hemorrhage, esophageal hemorrhage, large intestinal hemorrhage, rectal hemorrhage, melaena, duodenal ulcer hemorrhage, gastric ulcer hemorrhage, peptic ulcer hemorrhage, large intestinal ulcer hemorrhage, esophageal ulcer hemorrhage, and hematochezia. The end of treatment period was defined as the treatment period plus 28 days. | Day 1 to end of treatment period, approx. 7 years |
| Percentage of Participants With Significant Renal Dysfunction | Significant renal dysfunction was defined as a serum creatinine value ≥ 2 times upper limit of normal (ULN) at two consecutive assessments at least 7 days apart | Day 1 to end of treatment period, approx. 7 years |
| Percentage of Participants With Newly Occurring Moderate or Severe Neutropenia | Moderate or severe neutropenia was defined as neutrophil counts less than 1.0×10E9/L. | Day 1 to end of treatment period, approx. 7 years |
| Percentage of Participants With Newly Occurring Severe Thrombocytopenia | Severe thrombocytopenia was defined as platelets counts less than 50×10E9/L. | Day 1 to end of treatment period, approx. 7 years |
| Time to Study Drug Discontinuation Due to an AE or Laboratory Abnormality | As recorded on the Study Treatment Completion electronic Case Report Form (eCRF), date and reason given.Only participants for whom the reason for stopping study medication was entered as AE or laboratory abnormality were considered. This time to event endpoint was calculated as the date of study drug discontinuation due to an AE or laboratory abnormality minus date of randomization plus 1. Participants who did not discontinue study medication due to an AE or laboratory abnormality were censored at the date of study drug discontinuation. | Day 1 to end of treatment period, approx. 7 years |
| Greenwood Village |
| Colorado |
| United States |
| Willis-Knighton Cancer Center Dept of Onc | Shreveport | Louisiana | 71103 | United States |
| Henry Ford Hospital Henry Ford | Detroit | Michigan | 48202 | United States |
| Midwest Cancer Care Physicians MMCC | Kansas City | Missouri | 64131 | United States |
| Mercy Medical Research Institute SC | Manchester | Missouri | 63021 | United States |
| Glacier View Research Institute - Cancer SC | Kalispell | Montana | 59901 | United States |
| Hackensack University Medical Center Department of Research | Hackensack | New Jersey | 07601 | United States |
| University of Texas MD Anderson Cancer Center Dept of MD Anderson (16) | Houston | Texas | 77030 | United States |
| Cancer Care Centers of South Texas HOAST CCC of So.TX- MedicalCenter(2) | San Antonio | Texas | 78229 | United States |
| Swedish Cancer Institute Ballard Campus | Seattle | Washington | 98107 | United States |
| Novartis Investigative Site | Herston | Queensland | 4029 | Australia |
| Novartis Investigative Site | Plovdiv | 4002 | Bulgaria |
| Novartis Investigative Site | Sofia | 1407 | Bulgaria |
| Novartis Investigative Site | Varna | 9010 | Bulgaria |
| Novartis Investigative Site | Edmonton | Alberta | T6G 2B7 | Canada |
| Novartis Investigative Site | Winnipeg | Manitoba | R3E 0V9 | Canada |
| Novartis Investigative Site | Brampton | Ontario | L6R 3J7 | Canada |
| Novartis Investigative Site | St. Catharines | Ontario | L2S 0A9 | Canada |
| Novartis Investigative Site | Québec | Quebec | G1J 1Z4 | Canada |
| Novartis Investigative Site | Guangzhou | Guangdong | 51000 | China |
| Novartis Investigative Site | Wuhan | Hubei | 430022 | China |
| Novartis Investigative Site | Nanjing | Jiangsu | 210029 | China |
| Novartis Investigative Site | Suzhou | Jiangsu | 215006 | China |
| Novartis Investigative Site | Shanghai | Shanghai Municipality | 200437 | China |
| Novartis Investigative Site | Tianjin | Tianjin Municipality | 300020 | China |
| Novartis Investigative Site | Hangzhou | Zhejiang | 310003 | China |
| Novartis Investigative Site | Beijing | 100044 | China |
| Novartis Investigative Site | Jinan | 250012 | China |
| Novartis Investigative Site | Shanghai | 200025 | China |
| Novartis Investigative Site | Shanghai | 200233 | China |
| Novartis Investigative Site | Copenhagen | DK-2100 | Denmark |
| Novartis Investigative Site | Herlev | DK 2730 | Denmark |
| Novartis Investigative Site | Athens | GR | 115 27 | Greece |
| Novartis Investigative Site | Ioannina | GR | 455 00 | Greece |
| Novartis Investigative Site | Athens | 115 27 | Greece |
| Novartis Investigative Site | Pátrai | 265 00 | Greece |
| Novartis Investigative Site | Shatin, New Territories | Hong Kong | Hong Kong |
| Novartis Investigative Site | Hong Kong | Hong Kong |
| Novartis Investigative Site | Bologna | BO | 40138 | Italy |
| Novartis Investigative Site | Cagliari | CA | 09126 | Italy |
| Novartis Investigative Site | San Giovanni Rotondo | FG | 71013 | Italy |
| Novartis Investigative Site | Florence | FI | 50134 | Italy |
| Novartis Investigative Site | Messina | ME | 98125 | Italy |
| Novartis Investigative Site | Pescara | PE | 65124 | Italy |
| Novartis Investigative Site | Reggio Calabria | RC | 89124 | Italy |
| Novartis Investigative Site | Orbassano | TO | 10043 | Italy |
| Novartis Investigative Site | Kuching | Sarawak | 93586 | Malaysia |
| Novartis Investigative Site | Kuala Selangor | 68000 | Malaysia |
| Novartis Investigative Site | Mexico City | Mexico City | 02990 | Mexico |
| Novartis Investigative Site | Mexico City | Mexico City | 06726 | Mexico |
| Novartis Investigative Site | Auckland | 1309 | New Zealand |
| Novartis Investigative Site | Auckland | New Zealand |
| Novartis Investigative Site | Christchurch | 8001 | New Zealand |
| Novartis Investigative Site | Moscow | 125167 | Russia |
| Novartis Investigative Site | Rostov-on-Don | 344022 | Russia |
| Novartis Investigative Site | Basel | 4031 | Switzerland |
| Novartis Investigative Site | Zurich | 8091 | Switzerland |
| Novartis Investigative Site | Khon Kaen | THA | 40002 | Thailand |
| Novartis Investigative Site | Bangkok | 10330 | Thailand |
| Novartis Investigative Site | Bangkok | 10400 | Thailand |
| Novartis Investigative Site | Chiang Mai | 50200 | Thailand |
| Novartis Investigative Site | Glasgow | Scotland | G12 0YN | United Kingdom |
| Novartis Investigative Site | Birmingham | B15 2TH | United Kingdom |
| Novartis Investigative Site | Bournemouth | BH7 7DW | United Kingdom |
| Novartis Investigative Site | Cardiff | CF14 4XW | United Kingdom |
| Novartis Investigative Site | Exeter | EX2 5DW | United Kingdom |
| Novartis Investigative Site | Kent | DA2 8DA | United Kingdom |
| Novartis Investigative Site | Macclesfield | SK10 3BL | United Kingdom |
| Novartis Investigative Site | Nottingham | NG5 1PB | United Kingdom |
| Novartis Investigative Site | Oxford | OX3 7LJ | United Kingdom |
| FG001 | Placebo | 10 mg/kg/day (once daily) for the first 2 weeks of treatment, followed by 20 mg/kg/day (once daily) from Week 2 to End of Treatment. After 3 months of treatment at 20 mg/kg/day, the dose was allowed to be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on serum ferritin responses. When a target serum ferritin level was reached (usually between 500 and 1000 µg/L), the dose could be reduced by 50% to maintain the serum ferritin within the target range |
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| Safety Analysis Set | All randomized patients who received at least one dose of study medication |
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| Full Analysis Set | All patients to whom study treatment had been assigned by randomization |
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| Treated | Treated = Received study drug |
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| Untreated | Untreated = Did not receive study drug |
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| COMPLETED |
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| NOT COMPLETED |
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Full Analysis Set (FAS)
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| ID | Title | Description |
|---|---|---|
| BG000 | Deferasirox | 10 mg/kg/day (once daily) for the first 2 weeks of treatment, followed by 20 mg/kg/day (once daily) from Week 2 to End of Treatment. After 3 months of treatment at 20 mg/kg/day, the dose was allowed to be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on serum ferritin responses. When a target serum ferritin level was reached (usually between 500 and 1000 µg/L), the dose could be reduced by 50% to maintain the serum ferritin within the target range |
| BG001 | Placebo | 10 mg/kg/day (once daily) for the first 2 weeks of treatment, followed by 20 mg/kg/day (once daily) from Week 2 to End of Treatment. After 3 months of treatment at 20 mg/kg/day, the dose was allowed to be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on serum ferritin responses. When a target serum ferritin level was reached (usually between 500 and 1000 µg/L), the dose could be reduced by 50% to maintain the serum ferritin within the target range |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Number | participants |
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| MDS risk category | International Prognostic Scoring System (IPSS} | 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 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Event-free Survival | Event-free survival was defined as the time from the date of randomization to the date of the first documented non-fatal event (worsening cardiac function, hospitalization for congestive heart failure, liver function impairment, liver cirrhosis, transformation to AML, as defined in the protocol), or death, whichever occurred first. Participants who did not experience a non-fatal event as of the time of data cut-off (end of study), as well as participants who did not experience a non-fatal event and stopped study participation before the data cut-off, were censored as specified in the protocol. | FAS: the FAS comprised all patients to whom study treatment had been assigned by randomization. According to the intention to treat principle, patients were analyzed according to the study treatment and strata they were assigned to during the randomization procedure. | Posted | Median | 95% Confidence Interval | days | Day 1 to end of treatment period, approx. 7 years |
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| Secondary | Percentage of Participants With Hematologic Improvement (HI) in Terms of Erythroid Response | HI in terms of erythroid responses was assessed based on International Working Group (IWG) criteria, with improvement defined as follows:
| FAS: the FAS comprised all patients to whom study treatment had been assigned by randomization. According to the intention to treat principle, patients were analyzed according to the study treatment and strata they were assigned to during the randomization procedure. | Posted | Number | 95% Confidence Interval | percentage of participants | Day 1 to end of treatment period, approx. 7 years |
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| Secondary | Overall Survival | Overall survival was calculated as the date of death (irrespective of cause) minus date of randomization plus 1. | FAS: the FAS comprised all patients to whom study treatment had been assigned by randomization. According to the intention to treat principle, patients were analyzed according to the study treatment and strata they were assigned to during the randomization procedure. | Posted | Median | 95% Confidence Interval | days | Day 1 to end of treatment period, approx. 7.4 years |
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| Secondary | Percentage of Participants With Newly Occurring Hypothyroidism Compared to Baseline | As assessed by annual measurement of Thyroid Stimulating Hormone (TSH) and free T4. Hypothyroidism was defined as follows and is inclusive of:
| FAS: the FAS comprised all patients to whom study treatment had been assigned by randomization. According to the intention to treat principle, patients were analyzed according to the study treatment and strata they were assigned to during the randomization procedure. | Posted | Number | 95% Confidence Interval | percentage of participants | Day 1 to end of treatment period, approx. 7 years |
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| Secondary | Percentage of Participants With Worsening Glucose Metabolism Compared to Baseline | As assessed by an annual glucose tolerance test (OGTT). Worsening glucose metabolism was defined as an increase in glucose metabolism category (normal, impaired glucose metabolism, diabetes mellitus) based on the American Diabetes Association criteria (American Diabetes Association 2009) compared to the baseline result. The last available lab assessment date was used as the cut-off date for the analysis. | FAS: the FAS comprised all patients to whom study treatment had been assigned by randomization. According to the intention to treat principle, patients were analyzed according to the study treatment and strata they were assigned to during the randomization procedure. | Posted | Number | 95% Confidence Interval | percentage of participants | Day 1 to end of treatment period, approx. 7 years |
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| Secondary | Time to Disease Progression | Disease progression was defined as follows:
Disease progression was calculated as follows: Date of diagnosis of MDS progression or date of first diagnosis of AML, minus date of randomization plus 1. Participants who neither experienced MDS progression nor progression to AML were censored at the last contact date. | FAS: the FAS comprised all patients to whom study treatment had been assigned by randomization. According to the intention to treat principle, patients were analyzed according to the study treatment and strata they were assigned to during the randomization procedure. | Posted | Median | 95% Confidence Interval | days | Day 1 to end of treatment period, approx. 7 years |
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| Secondary | Time to First Occurrence of Serum Ferritin Level >2 Times the Baseline Value at Two Consecutive Assessments (at Least Two Weeks Apart) | Assessed by blood draw and calculated as follows: Date of first occurrence of serum ferritin >2 times the baseline value at two consecutive assessments (at least two weeks apart), minus date of randomization plus 1. Participants who did not experience such an increase were censored at the last date when serum ferritin was available. | FAS: the FAS comprised all patients to whom study treatment had been assigned by randomization. According to the intention to treat principle, patients were analyzed according to the study treatment and strata they were assigned to during the randomization procedure. | Posted | Median | 95% Confidence Interval | days | Day 1 to end of treatment period, approx. 7 years |
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| Secondary | Time to at Least a 10% Increase From Baseline in Left Ventricular End-diastolic Internal (LVIDD) at Two Consecutive Assessments at Least Two Weeks Apart | Assessed by echocardiography and calculated as follows: Date of echocardiography assessment where a minimum of 10% increase of LVIDD first occurred, minus date of randomization plus 1. Participants who did not experience such an increase were censored at the last date when LVIDD was available. | FAS: the FAS comprised all patients to whom study treatment had been assigned by randomization. According to the intention to treat principle, patients were analyzed according to the study treatment and strata they were assigned to during the randomization procedure. | Posted | Median | 95% Confidence Interval | days | Day 1 to end of treatment period, approx. 7 years |
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| Secondary | Time to at Least a 10% Increase From Baseline in Left Ventricular Internal Systolic Diameter (LVISD) at Two Consecutive Assessments at Least Two Weeks Apart | Assessed by echocardiography and calculated as follows: Date of echocardiography assessment where a minimum of 10% increase of LVISD first occurred, minus date of randomization plus 1. Participants who did not experience such an increase were censored at the last date when LVISD was available. | FAS: the FAS comprised all patients to whom study treatment had been assigned by randomization. According to the intention to treat principle, patients were analyzed according to the study treatment and strata they were assigned to during the randomization procedure. | Posted | Median | 95% Confidence Interval | days | Day 1 to end of treatment period, approx. 7 years |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Total Number of Infections Requiring Intravenous Antimicrobials | The total number of infections were counted and summarized per treatment group. For this number, one participant can contribute more than one infection event. Infections were determined from the reported AEs with system organ class "Infections and infestations" and action taken "Concomitant medication taken." Antimicrobial therapy was determined from the reported concomitant medications for participants who had an infection AE. The route of administration needed to be specified as "intravenous (i.v.)". End of treatment period was defined as the treatment period plus 28 days. | SAS: the safety set included all randomized patients who received at least one dose of study medication. Patients were analyzed according to the study treatment they actually received and the strata they were assigned. No Statistical Analysis was performed | Posted | Number | infections | Day 1 to end of treatment period, approx. 7 years |
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| Secondary | Percentage of Participants With Major Gastrointestinal Bleeding | Major gastrointestinal bleeding was defined as an AE that could include one of the following MedDRA preferred terms: gastric hemorrhage, gastrointestinal hemorrhage, small intestinal hemorrhage, esophageal hemorrhage, large intestinal hemorrhage, rectal hemorrhage, melaena, duodenal ulcer hemorrhage, gastric ulcer hemorrhage, peptic ulcer hemorrhage, large intestinal ulcer hemorrhage, esophageal ulcer hemorrhage, and hematochezia. The end of treatment period was defined as the treatment period plus 28 days. | SAS: the safety set included all randomized patients who received at least one dose of study medication. Patients were analyzed according to the study treatment they actually received and the strata they were assigned. | Posted | Number | 95% Confidence Interval | percentage of participants | Day 1 to end of treatment period, approx. 7 years |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Significant Renal Dysfunction | Significant renal dysfunction was defined as a serum creatinine value ≥ 2 times upper limit of normal (ULN) at two consecutive assessments at least 7 days apart | SAS: the safety set included all randomized patients who received at least one dose of study medication. Patients were analyzed according to the study treatment they actually received and the strata they were assigned. | Posted | Number | 95% Confidence Interval | percentage of participants | Day 1 to end of treatment period, approx. 7 years |
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| Secondary | Percentage of Participants With Newly Occurring Moderate or Severe Neutropenia | Moderate or severe neutropenia was defined as neutrophil counts less than 1.0×10E9/L. | SAS: the safety set included all randomized patients who received at least one dose of study medication. Patients were analyzed according to the study treatment they actually received and the strata they were assigned. | Posted | Number | 95% Confidence Interval | percentage of participants | Day 1 to end of treatment period, approx. 7 years |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Newly Occurring Severe Thrombocytopenia | Severe thrombocytopenia was defined as platelets counts less than 50×10E9/L. | SAS: the safety set included all randomized patients who received at least one dose of study medication. Patients were analyzed according to the study treatment they actually received and the strata they were assigned. | Posted | Number | 95% Confidence Interval | percentage of participants | Day 1 to end of treatment period, approx. 7 years |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Time to Study Drug Discontinuation Due to an AE or Laboratory Abnormality | As recorded on the Study Treatment Completion electronic Case Report Form (eCRF), date and reason given.Only participants for whom the reason for stopping study medication was entered as AE or laboratory abnormality were considered. This time to event endpoint was calculated as the date of study drug discontinuation due to an AE or laboratory abnormality minus date of randomization plus 1. Participants who did not discontinue study medication due to an AE or laboratory abnormality were censored at the date of study drug discontinuation. | SAS: the safety set included all randomized patients who received at least one dose of study medication. Patients were analyzed according to the study treatment they actually received and the strata they were assigned. | Posted | Median | 95% Confidence Interval | days | Day 1 to end of treatment period, approx. 7 years |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Post-Hoc | All Collected Deaths | On-treatment deaths were collected from FPFT up to 28 days after study drug discontinuation, for a maximum duration of 2627 days (2599 plus 28 days) (treatment duration ranged from 1 day to 2599 days). Deaths post treatment survival follow up were collected after the on treatment period, up to approx. 7.4 years. Patients who had not experienced any of the non-fatal events from the composite primary endpoint and had not stopped study participation at the time of data cut-off (end of study) were censored. | FAS: the FAS comprised all patients to whom study treatment had been assigned by randomization. According to the intention to treat principle, patients were analyzed according to the study treatment and strata they were assigned to during the randomization procedure. | Posted | Number | Participants | 2627 days, approx. 7.4 years |
|
On treatment deaths were collected from FPFT up to 28 days after study drug discontinuation, for a maximum duration of 2627 days (2599 plus 28 days) (treatment duration ranged from 1 day to 2599 days).
Any sign or symptom that occurs during the study treatment plus 28 days post treatment
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Deferasirox | 10 mg/kg/day (once daily) for the first 2 weeks of treatment, followed by 20 mg/kg/day (once daily) from Week 2 to End of Treatment. After 3 months of treatment at 20 mg/kg/day, the dose was allowed to be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on serum ferritin responses. When a target serum ferritin level was reached (usually between 500 and 1000 µg/L), the dose could be reduced by 50% to maintain the serum ferritin within the target range | 24 | 148 | 80 | 148 | 135 | 148 |
| EG001 | Placebo | 10 mg/kg/day (once daily) for the first 2 weeks of treatment, followed by 20 mg/kg/day (once daily) from Week 2 to End of Treatment. After 3 months of treatment at 20 mg/kg/day, the dose was allowed to be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on serum ferritin responses. When a target serum ferritin level was reached (usually between 500 and 1000 µg/L), the dose could be reduced by 50% to maintain the serum ferritin within the target range | 10 | 76 | 38 | 76 | 65 | 76 |
| EG002 | All Patients | Combined patients from the Deferasirox and Placebo arms | 34 | 224 | 118 | 224 | 200 | 224 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal lymphadenopathy | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Bone marrow failure | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cytopenia | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Lymphadenitis | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Normochromic normocytic anaemia | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Splenic infarction | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Splenic lesion | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Splenomegaly | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cardio-respiratory arrest | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cardiomyopathy | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cardiopulmonary failure | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cardiovascular insufficiency | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Left ventricular failure | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Right ventricular dysfunction | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Sinus node dysfunction | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Aural polyp | Ear and labyrinth disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hypogonadism | Endocrine disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Retinal haemorrhage | Eye disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Vitreous floaters | Eye disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Ascites | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Crohn's disease | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Gastrointestinal perforation | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Gingival hypertrophy | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Haematemesis | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Haemorrhoidal haemorrhage | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hiatus hernia | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Intestinal mass | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Face oedema | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| General physical health deterioration | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Multiple organ dysfunction syndrome | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Oedema | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hepatic function abnormal | Hepatobiliary disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hepatic lesion | Hepatobiliary disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hypogammaglobulinaemia | Immune system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Abscess soft tissue | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Anal abscess | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Anal infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Appendiceal abscess | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Clostridial infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Diarrhoea infectious | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Escherichia sepsis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Escherichia urinary tract infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Febrile infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Gingivitis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Groin abscess | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Liver abscess | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Lung infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Neutropenic sepsis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Orchitis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Peritonitis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Scrub typhus | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Skin graft infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Soft tissue infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Tongue fungal infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Back injury | Injury, poisoning and procedural complications | MedDRA (20.1) | Systematic Assessment |
| |
| Face injury | Injury, poisoning and procedural complications | MedDRA (20.1) | Systematic Assessment |
| |
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA (20.1) | Systematic Assessment |
| |
| Hip fracture | Injury, poisoning and procedural complications | MedDRA (20.1) | Systematic Assessment |
| |
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA (20.1) | Systematic Assessment |
| |
| Pelvic fracture | Injury, poisoning and procedural complications | MedDRA (20.1) | Systematic Assessment |
| |
| Rib fracture | Injury, poisoning and procedural complications | MedDRA (20.1) | Systematic Assessment |
| |
| Soft tissue injury | Injury, poisoning and procedural complications | MedDRA (20.1) | Systematic Assessment |
| |
| Splenic rupture | Injury, poisoning and procedural complications | MedDRA (20.1) | Systematic Assessment |
| |
| Subdural haematoma | Injury, poisoning and procedural complications | MedDRA (20.1) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (20.1) | Systematic Assessment |
| |
| Blood creatine increased | Investigations | MedDRA (20.1) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (20.1) | Systematic Assessment |
| |
| Blood pressure decreased | Investigations | MedDRA (20.1) | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA (20.1) | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (20.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Mobility decreased | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Muscle atrophy | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Sjogren's syndrome | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Synovial cyst | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Acute leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.1) | Systematic Assessment |
| |
| Acute myeloid leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.1) | Systematic Assessment |
| |
| Bladder neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.1) | Systematic Assessment |
| |
| Light chain disease | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.1) | Systematic Assessment |
| |
| Myelodysplastic syndrome | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.1) | Systematic Assessment |
| |
| Myelofibrosis | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (20.1) | Systematic Assessment |
| |
| Cerebral haematoma | Nervous system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cerebral haemorrhage | Nervous system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cognitive disorder | Nervous system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Coma | Nervous system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hemianopia homonymous | Nervous system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Anuria | Renal and urinary disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Nephropathy toxic | Renal and urinary disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Benign prostatic hyperplasia | Reproductive system and breast disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Uterine haemorrhage | Reproductive system and breast disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Uterine polyp | Reproductive system and breast disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Interstitial lung disease | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Pulmonary fibrosis | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Pulmonary haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Pulmonary mass | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Aortic stenosis | Vascular disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (20.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (20.1) | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (20.1) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (20.1) | Systematic Assessment |
| |
| Creatinine renal clearance decreased | Investigations | MedDRA (20.1) | Systematic Assessment |
| |
| Protein urine present | Investigations | MedDRA (20.1) | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (20.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (20.1) | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-8300 | novartis.email@novartis.com |
| SAP_000.pdf |
| Prot | Yes | No | No | Study Protocol | Sep 22, 2014 | Feb 25, 2019 | Prot_001.pdf |
| ID | Term |
|---|---|
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| 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 |
Not provided
Not provided
| Male |
|
| Chinese |
|
| Mixed ethnicity |
|
| Other |
|
| Intermediate 1 (combined score 0.5 - 1.0) |
|
10 mg/kg/day (once daily) for the first 2 weeks of treatment, followed by 20 mg/kg/day (once daily) from Week 2 to End of Treatment. After 3 months of treatment at 20 mg/kg/day, the dose was allowed to be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on serum ferritin responses. When a target serum ferritin level was reached (usually between 500 and 1000 µg/L), the dose could be reduced by 50% to maintain the serum ferritin within the target range
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10 mg/kg/day (once daily) for the first 2 weeks of treatment, followed by 20 mg/kg/day (once daily) from Week 2 to End of Treatment. After 3 months of treatment at 20 mg/kg/day, the dose was allowed to be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on serum ferritin responses. When a target serum ferritin level was reached (usually between 500 and 1000 µg/L), the dose could be reduced by 50% to maintain the serum ferritin within the target range
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10 mg/kg/day (once daily) for the first 2 weeks of treatment, followed by 20 mg/kg/day (once daily) from Week 2 to End of Treatment. After 3 months of treatment at 20 mg/kg/day, the dose was allowed to be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on serum ferritin responses. When a target serum ferritin level was reached (usually between 500 and 1000 µg/L), the dose could be reduced by 50% to maintain the serum ferritin within the target range
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