Clinical Trial Results:
A phase II, multi-center, single-arm, prospective study to evaluate the safety and efficacy of deferasirox in beta-thalassemia major patients after hematopoietic stem cell transplantation
Summary
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EudraCT number |
2016-001561-88 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
21 Oct 2015
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Results information
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Results version number |
v1(current) |
This version publication date |
25 Jun 2016
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First version publication date |
25 Jun 2016
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Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
CICL670ATR04
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT01610297 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Novartis
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Sponsor organisation address |
CH-4002, Basel, Switzerland,
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Public contact |
Clinical Disclosure Office, Novartis Pharma AG, 41 613241111,
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Scientific contact |
Clinical Disclosure Office, Novartis Pharma AG, 41 613241111,
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
21 Oct 2015
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
21 Oct 2015
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The primary objective of the present study was to determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major in 12 months period
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Protection of trial subjects |
The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
19 Sep 2013
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Turkey: 27
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Worldwide total number of subjects |
27
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EEA total number of subjects |
0
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
20
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Adolescents (12-17 years) |
7
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
- | ||||||||||
Pre-assignment
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Screening details |
The patients received oral deferasirox at an initial dose of 10 mg/kg/day and dose escalation was allowed up to 20 mg/kg daily for 12 months or until the serum ferritin level was below 500 μg/L. Dose titration was allowed in 3 months periods by 5 mg/kg/day at the discretion of the investigator | ||||||||||
Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||
Arms
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Arm title
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ICL670 | ||||||||||
Arm description |
Oral dose of ICL670 at 10 mg/kg daily | ||||||||||
Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
Deferasinox
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Investigational medicinal product code |
ICL670
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Other name |
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Pharmaceutical forms |
Dispersible tablet
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Routes of administration |
Oral use
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Dosage and administration details |
deferasirox (ICL670) 10 mg/kg, oral, daily
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Baseline characteristics reporting groups
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Reporting group title |
ICL670
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Reporting group description |
Oral dose of ICL670 at 10 mg/kg daily | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
ICL670
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Reporting group description |
Oral dose of ICL670 at 10 mg/kg daily |
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End point title |
Number of Participants with Adverse Events, Serious Adverse Events and Deaths as a measure of Safety and Tolerability [1] | ||||||||||||
End point description |
To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major in 12 months period. No statistical analysis was planned for this primary outcome. The Safety Set (SS) includes all included patients who were included in the study.
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End point type |
Primary
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End point timeframe |
12 months
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Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: There was no planned analysis for this safety endpoint. |
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No statistical analyses for this end point |
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End point title |
Change in serum ferritin level | ||||||||||||
End point description |
Friedman test and Wilcoxon Signed Rank test were conducted to test whether there is a significant change in the serum ferritin level. The The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose.
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End point type |
Secondary
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End point timeframe |
Baseline, 12 Months
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No statistical analyses for this end point |
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End point title |
Change in the further parameters of iron overload (cardiac iron concentration by MR examination) | ||||||||||||
End point description |
Friedman test and Wilcoxon Signed Rank test were conducted to test whether there is a significant change in the T2*MRI (Cardiac MRI). The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose.
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End point type |
Secondary
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End point timeframe |
Baseline, 12 month
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No statistical analyses for this end point |
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End point title |
The percentage of patients reaching serum ferritin levels lower than 500 μg/L | ||||||||||||
End point description |
Friedman test and Wilcoxon Signed Rank test were conducted to test whether there is a significant change in the serum ferritin level. The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose.
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End point type |
Secondary
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End point timeframe |
Week 28 and Week 52
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No statistical analyses for this end point |
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End point title |
Change in the further parameters of iron overload (liver iron concentration by Magnetic resonance imaging (MRI examination) | ||||||||||||
End point description |
Friedman test and Wilcoxon Signed Rank test were conducted to test whether there is a significant change in the R2*MRI (Liver MRI).
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End point type |
Secondary
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End point timeframe |
Baseline, 12 Months
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No statistical analyses for this end point |
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Adverse events information
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Timeframe for reporting adverse events |
Adverse Events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All Adverse events are reported in this record from First Patient First Treatment until Last Patient Last Visit.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
18.0
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Reporting groups
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Reporting group title |
ICL670
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Reporting group description |
Oral dose of ICL670 at 10 mg/kg daily | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
None reported |