Clinical Trial Results:
An extension study of eltrombopag in pediatric patients with chronic immune (idiopathic) thrombocytopenia purpura (ITP)
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Summary
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EudraCT number |
2017-004082-27 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
04 Jul 2017
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Results information
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Results version number |
v1(current) |
This version publication date |
24 Feb 2018
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First version publication date |
24 Feb 2018
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Other versions |
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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 |
117366
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT02201290 | ||
WHO universal trial number (UTN) |
- | ||
Other trial identifiers |
CETB115BRU01: 2017-004082-27 | ||
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Sponsors
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Sponsor organisation name |
Novartis Pharma, AG
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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, novartis.email@novartis.com
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Scientific contact |
Clinical Disclosure Office, Novartis Pharma, AG, 41 613241111, novartis.email@novartis.com
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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 |
12 Dec 2017
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
04 Jul 2017
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Global end of trial reached? |
Yes
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Global end of trial date |
04 Jul 2017
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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 main objective was to provide continued treatment to patients who have completed the TRA115450 study and to describe the safety and tolerability of Eltrombopag when administered to pediatric subjects with previously treated chronic ITP.
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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 |
18 Jun 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 |
Russian Federation: 9
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Worldwide total number of subjects |
9
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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) |
6
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Adolescents (12-17 years) |
3
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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 |
The study included pediatric patients with chronic ITP. | ||||||||||||||
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Pre-assignment
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Screening details |
Study screening period was followed by a single arm treatment period and a follow-up period | ||||||||||||||
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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 |
Non-randomised - controlled
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Blinding used |
Not blinded | ||||||||||||||
Blinding implementation details |
This was an open-label study.
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Arms
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Arm title
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Treated participants | ||||||||||||||
Arm description |
- | ||||||||||||||
Arm type |
Experimental | ||||||||||||||
Investigational medicinal product name |
Eltrombopag (CETB115B)
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Investigational medicinal product code |
Eltrombopag tablets
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Other name |
CETB115B
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
40mg
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Baseline characteristics reporting groups
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Reporting group title |
Treated participants
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
ATS
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Subject analysis set type |
Safety analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
The all treated subjects (ATS) analysis set included all patients who received at least one dose of Eltrombopag.
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End points reporting groups
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Reporting group title |
Treated participants
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Reporting group description |
- | ||
Subject analysis set title |
ATS
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
The all treated subjects (ATS) analysis set included all patients who received at least one dose of Eltrombopag.
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End point title |
Adverse Events [1] | |||||||||||||||||||||||||||||||||
End point description |
Frequency of all adverse events (including Ophthalmic events) categorized using CTCAE toxicity grades and clinical laboratory test [ Time Frame: Up to Week 4 Follow-up period ] Clinical laboratory assessments and frequency of all adverse events, categorized using Common Terminology Criteria for Adverse Events (CTCAE) toxicity grades will present safety and tolerability endpoints
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End point type |
Primary
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End point timeframe |
Up to week 4 follow up period
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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: Stats analysis does not apply to this endpoint |
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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 reported in this record are from date of First Patient First Treatment until Last Patient Last Visit
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
19.0
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Reporting groups
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Reporting group title |
Eltrombopag
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Reporting group description |
Eltrombopag | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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? Yes | |||
Date |
Amendment |
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08 Jul 2013 |
Protocol was amended 1 time. Previous sections of this report describe the study conduct as amended. The key features of amendment are given below:
Amendment 01 (08-Jul-2013) introduced the following changes:
Sponsor Medical Monitor was updated.
The rationale of the study was clarified and the study assessments simplified
Clarification of the term ‘study treatment’.
Clarification of dosing by age rather than cohort. Additional guidance was included for those subjects who switched formulation during the study.
Clarification around the time period to record prior medications.
Correction of study number within table as well as clarification of assessments.
Clarification of endpoints.
Clarification consistent with revised assessments and match Time and Events Table.
Clarification physical examination and the removal of vital signs.
Renal assessments were removed.
Title was changed to reflect more streamlined assessment and focus on cataract assessment. Subsections 7.2.1.5.1. Baseline, Ocular History and Risk Factors, 7.2.1.5.2, Ophthalmic Examinations and 7.2.1.5.4 Additional Ocular Follow-up were removed.
Laboratory assessments were streamlined. Table 4 was amended to reflect this change.
Clarification around administration of Eltrombopag.
Clarification to data process and removal of repeated text.
The Reporting and Analysis Plan was removed. This section and each sub-section within were changed for the modification of analysis and data collection. These amendments were not considered to have affected the interpretation of study results. |
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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 | |||
