The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00940602




Registration number
NCT00940602
Ethics application status
Date submitted
15/07/2009
Date registered
16/07/2009

Titles & IDs
Public title
Myelodysplastic Syndromes (MDS) Event Free Survival With Iron Chelation Therapy Study
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
Secondary ID [1] 0 0
2009-012418-38
Secondary ID [2] 0 0
CICL670A2302
Universal Trial Number (UTN)
Trial acronym
TELESTO
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelodysplastic Syndromes 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
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

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Event-free Survival
Timepoint [1] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [1] 0 0
Percentage of Participants With Hematologic Improvement (HI) in Terms of Erythroid Response
Timepoint [1] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [2] 0 0
Overall Survival
Timepoint [2] 0 0
Day 1 to end of treatment period, approx. 7.4 years
Secondary outcome [3] 0 0
Percentage of Participants With Newly Occurring Hypothyroidism Compared to Baseline
Timepoint [3] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [4] 0 0
Percentage of Participants With Worsening Glucose Metabolism Compared to Baseline
Timepoint [4] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [5] 0 0
Time to Disease Progression
Timepoint [5] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [6] 0 0
Time to First Occurrence of Serum Ferritin Level >2 Times the Baseline Value at Two Consecutive Assessments (at Least Two Weeks Apart)
Timepoint [6] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [7] 0 0
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
Timepoint [7] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [8] 0 0
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
Timepoint [8] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [9] 0 0
Total Number of Infections Requiring Intravenous Antimicrobials
Timepoint [9] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [10] 0 0
Percentage of Participants With Major Gastrointestinal Bleeding
Timepoint [10] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [11] 0 0
Percentage of Participants With Significant Renal Dysfunction
Timepoint [11] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [12] 0 0
Percentage of Participants With Newly Occurring Moderate or Severe Neutropenia
Timepoint [12] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [13] 0 0
Percentage of Participants With Newly Occurring Severe Thrombocytopenia
Timepoint [13] 0 0
Day 1 to end of treatment period, approx. 7 years
Secondary outcome [14] 0 0
Time to Study Drug Discontinuation Due to an AE or Laboratory Abnormality
Timepoint [14] 0 0
Day 1 to end of treatment period, approx. 7 years

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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Novartis Investigative Site - Herston
Recruitment postcode(s) [1] 0 0
4029 - Herston
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Louisiana
Country [4] 0 0
United States of America
State/province [4] 0 0
Michigan
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
Montana
Country [7] 0 0
United States of America
State/province [7] 0 0
New Jersey
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
United States of America
State/province [9] 0 0
Washington
Country [10] 0 0
Bulgaria
State/province [10] 0 0
Plovdiv
Country [11] 0 0
Bulgaria
State/province [11] 0 0
Sofia
Country [12] 0 0
Bulgaria
State/province [12] 0 0
Varna
Country [13] 0 0
Canada
State/province [13] 0 0
Alberta
Country [14] 0 0
Canada
State/province [14] 0 0
Manitoba
Country [15] 0 0
Canada
State/province [15] 0 0
Ontario
Country [16] 0 0
Canada
State/province [16] 0 0
Quebec
Country [17] 0 0
China
State/province [17] 0 0
Guangdong
Country [18] 0 0
China
State/province [18] 0 0
Hubei
Country [19] 0 0
China
State/province [19] 0 0
Jiangsu
Country [20] 0 0
China
State/province [20] 0 0
Shanghai
Country [21] 0 0
China
State/province [21] 0 0
Tianjin
Country [22] 0 0
China
State/province [22] 0 0
Zhejiang
Country [23] 0 0
China
State/province [23] 0 0
Beijing
Country [24] 0 0
China
State/province [24] 0 0
Jinan
Country [25] 0 0
Denmark
State/province [25] 0 0
Copenhagen
Country [26] 0 0
Denmark
State/province [26] 0 0
Herlev
Country [27] 0 0
Greece
State/province [27] 0 0
GR
Country [28] 0 0
Greece
State/province [28] 0 0
Athens
Country [29] 0 0
Greece
State/province [29] 0 0
Patras
Country [30] 0 0
Hong Kong
State/province [30] 0 0
Hong Kong
Country [31] 0 0
Hong Kong
State/province [31] 0 0
Shatin, New Territories
Country [32] 0 0
Italy
State/province [32] 0 0
BO
Country [33] 0 0
Italy
State/province [33] 0 0
CA
Country [34] 0 0
Italy
State/province [34] 0 0
FG
Country [35] 0 0
Italy
State/province [35] 0 0
FI
Country [36] 0 0
Italy
State/province [36] 0 0
ME
Country [37] 0 0
Italy
State/province [37] 0 0
PE
Country [38] 0 0
Italy
State/province [38] 0 0
RC
Country [39] 0 0
Italy
State/province [39] 0 0
TO
Country [40] 0 0
Malaysia
State/province [40] 0 0
Sarawak
Country [41] 0 0
Malaysia
State/province [41] 0 0
Selangor
Country [42] 0 0
Mexico
State/province [42] 0 0
Distrito Federal
Country [43] 0 0
New Zealand
State/province [43] 0 0
Auckland 6
Country [44] 0 0
New Zealand
State/province [44] 0 0
Auckland
Country [45] 0 0
New Zealand
State/province [45] 0 0
Christchurch
Country [46] 0 0
Russian Federation
State/province [46] 0 0
Moscow
Country [47] 0 0
Russian Federation
State/province [47] 0 0
Rostov on Don
Country [48] 0 0
Switzerland
State/province [48] 0 0
Basel
Country [49] 0 0
Switzerland
State/province [49] 0 0
Zurich
Country [50] 0 0
Thailand
State/province [50] 0 0
THA
Country [51] 0 0
Thailand
State/province [51] 0 0
Bangkok
Country [52] 0 0
Thailand
State/province [52] 0 0
Chiang Mai
Country [53] 0 0
United Kingdom
State/province [53] 0 0
Scotland
Country [54] 0 0
United Kingdom
State/province [54] 0 0
Birmingham
Country [55] 0 0
United Kingdom
State/province [55] 0 0
Bournemouth
Country [56] 0 0
United Kingdom
State/province [56] 0 0
Cardiff
Country [57] 0 0
United Kingdom
State/province [57] 0 0
Exeter
Country [58] 0 0
United Kingdom
State/province [58] 0 0
Kent
Country [59] 0 0
United Kingdom
State/province [59] 0 0
Macclesfield
Country [60] 0 0
United Kingdom
State/province [60] 0 0
Nottingham
Country [61] 0 0
United Kingdom
State/province [61] 0 0
Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: http://www.clinicalstudydatarequest.com


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.