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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT00940602
Registration number
NCT00940602
Ethics application status
Date submitted
15/07/2009
Date registered
16/07/2009
Date last updated
23/11/2020
Titles & IDs
Public title
Myelodysplastic Syndromes (MDS) Event Free Survival With Iron Chelation Therapy Study
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Scientific title
A Multi-center, Randomized, Double-blind, Placebo-controlled Clinical Trial of Deferasirox in Patients With Myelodysplastic Syndromes (Low/Int-1 Risk) and Transfusional Iron Overload
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Secondary ID [1]
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2009-012418-38
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Secondary ID [2]
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CICL670A2302
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Universal Trial Number (UTN)
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Trial acronym
TELESTO
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Myelodysplastic Syndromes
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Condition category
Condition code
Blood
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Haematological diseases
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Blood
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Other blood disorders
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Deferasirox
Treatment: Drugs - Placebo
Experimental: 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.
Placebo Comparator: 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.
Treatment: Drugs: Deferasirox
Deferasirox provided as 125 mg, 250 mg, and 500 mg dispersible tablets for oral use
Treatment: Drugs: Placebo
Inactive ingredients used as a placebo comparator, provided as 125 mg, 250 mg, and 500 mg dispersible tablets for oral use
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Event-free Survival
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Assessment method [1]
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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.
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Timepoint [1]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [1]
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Percentage of Participants With Hematologic Improvement (HI) in Terms of Erythroid Response
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Assessment method [1]
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HI in terms of erythroid responses was assessed based on International Working Group (IWG) criteria, with improvement defined as follows:
Hemoglobin increase of = 1.5 g/dL OR
Reduction of = 4 RBC transfusions/8 weeks in comparison to pre-treatment values and lasting at least 8 weeks. The last hemoglobin value measured prior to randomization was used as the pre-treatment value. The last available lab assessment date was used as the cut-off date for the analysis.
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Timepoint [1]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [2]
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Overall Survival
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Assessment method [2]
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Overall survival was calculated as the date of death (irrespective of cause) minus date of randomization plus 1.
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Timepoint [2]
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Day 1 to end of treatment period, approx. 7.4 years
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Secondary outcome [3]
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Percentage of Participants With Newly Occurring Hypothyroidism Compared to Baseline
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Assessment method [3]
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As assessed by annual measurement of Thyroid Stimulating Hormone (TSH) and free T4. Hypothyroidism was defined as follows and is inclusive of:
Primary hypothyroidism: serum TSH >upper limit of normal (ULN) and free T4 <lower limit of normal (LLN);
Secondary hypothyroidism: serum TSH <ULN and free T4 <lower limit of normal;
Subclinical hypothyroidism: TSH >ULN and a free T4 within normal limits. The last available lab assessment date was used as the cut-off date for the analysis.
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Timepoint [3]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [4]
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Percentage of Participants With Worsening Glucose Metabolism Compared to Baseline
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Assessment method [4]
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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.
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Timepoint [4]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [5]
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Time to Disease Progression
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Assessment method [5]
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Disease progression was defined as follows:
MDS progression: Transition into a higher MDS risk group based on IPSS scoring
Progression to AML: 20 percent or more blasts seen in the bone marrow collected by biopsy or aspirate.
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.
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Timepoint [5]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [6]
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Time to First Occurrence of Serum Ferritin Level >2 Times the Baseline Value at Two Consecutive Assessments (at Least Two Weeks Apart)
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Assessment method [6]
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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.
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Timepoint [6]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [7]
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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
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Assessment method [7]
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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.
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Timepoint [7]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [8]
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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
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Assessment method [8]
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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.
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Timepoint [8]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [9]
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Total Number of Infections Requiring Intravenous Antimicrobials
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Assessment method [9]
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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.
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Timepoint [9]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [10]
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Percentage of Participants With Major Gastrointestinal Bleeding
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Assessment method [10]
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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.
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Timepoint [10]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [11]
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Percentage of Participants With Significant Renal Dysfunction
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Assessment method [11]
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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
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Timepoint [11]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [12]
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Percentage of Participants With Newly Occurring Moderate or Severe Neutropenia
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Assessment method [12]
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Moderate or severe neutropenia was defined as neutrophil counts less than 1.0×10E9/L.
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Timepoint [12]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [13]
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Percentage of Participants With Newly Occurring Severe Thrombocytopenia
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Assessment method [13]
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Severe thrombocytopenia was defined as platelets counts less than 50×10E9/L.
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Timepoint [13]
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Day 1 to end of treatment period, approx. 7 years
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Secondary outcome [14]
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Time to Study Drug Discontinuation Due to an AE or Laboratory Abnormality
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Assessment method [14]
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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.
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Timepoint [14]
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Day 1 to end of treatment period, approx. 7 years
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Eligibility
Key inclusion criteria
- Weigh between 35-135 kilograms
- Low or int-1 risk MDS
- Ferritin >1000 micrograms/liter at screening
- History of transfusion of 15 to 75 Packed Red Blood Cells (PRBC) units
- Anticipated to be transfused with at least 8 units of PRBCs annually during the study
- Women of child-bearing potential using effective methods of contraception during
dosing of study treatment
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- More than 6 months of cumulative ICT (such as daily deferasirox (Exjade®) or
deferiprone or 5×/week deferoxamine)
- More than 3 years since patient began receiving regular transfusions (2 units per 8
weeks or 4 units received in a 3 month period)
- Significant proteinuria
- History of hospitalization for congestive heart failure; other heart conditions as
specified in the protocol
- Systemic diseases which would prevent study treatment
- Hepatitis B; Hepatitis C; HIV
- Liver cirrhosis
- Pregnant, or breast-feeding patients, or patients of child-bearing potential not
employing an effective method of birth control
- History of drug or alcohol abuse within the 12 months prior to enrollment
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
22/03/2010
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
27/02/2018
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Sample size
Target
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Accrual to date
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Final
225
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Recruitment in Australia
Recruitment state(s)
QLD
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Recruitment hospital [1]
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Novartis Investigative Site - Herston
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Recruitment postcode(s) [1]
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4029 - Herston
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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United States of America
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State/province [2]
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Colorado
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United States of America
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Louisiana
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United States of America
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Michigan
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Country [5]
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United States of America
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Missouri
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United States of America
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Montana
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United States of America
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New Jersey
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United States of America
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Texas
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United States of America
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Washington
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Bulgaria
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State/province [10]
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Plovdiv
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Bulgaria
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State/province [11]
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Sofia
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Bulgaria
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State/province [12]
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Varna
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Canada
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Alberta
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Canada
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Manitoba
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Canada
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Ontario
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Canada
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Quebec
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China
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Guangdong
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China
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Hubei
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China
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Jiangsu
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China
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Shanghai
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China
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Tianjin
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China
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Zhejiang
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China
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Beijing
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China
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Jinan
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Denmark
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Copenhagen
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Denmark
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Herlev
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Greece
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GR
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Greece
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Athens
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Greece
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Patras
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Hong Kong
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Hong Kong
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Hong Kong
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State/province [31]
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Shatin, New Territories
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Italy
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BO
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Italy
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CA
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Italy
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FG
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Italy
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FI
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Italy
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ME
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Italy
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PE
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Italy
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RC
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Italy
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TO
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Country [40]
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Malaysia
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Sarawak
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Malaysia
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Selangor
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Mexico
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Distrito Federal
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New Zealand
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State/province [43]
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Auckland 6
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New Zealand
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Auckland
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New Zealand
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Christchurch
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Russian Federation
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Moscow
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Russian Federation
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Rostov on Don
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Switzerland
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Basel
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Switzerland
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Zurich
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Thailand
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THA
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Thailand
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Bangkok
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Thailand
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Chiang Mai
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United Kingdom
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Scotland
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United Kingdom
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Birmingham
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United Kingdom
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Bournemouth
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United Kingdom
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Cardiff
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United Kingdom
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Exeter
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United Kingdom
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Kent
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United Kingdom
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Macclesfield
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United Kingdom
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Nottingham
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United Kingdom
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State/province [61]
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Oxford
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Novartis Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT00940602
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Novartis Pharmaceuticals
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Address
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Novartis Pharmaceuticals
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Country
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Phone
0
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Fax
0
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Email
0
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Contact person for public queries
Name
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Address
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT00940602
Download to PDF