Clinical Trial Results:
A 48-week, Open-label, 2-arm, Parallel-group, Randomized Exploratory Study to Assess Liver Iron Concentration Measured by FerriScan® (R2) Magnetic Resonance Imaging in Beta-thalassemia Subjects Administered SPD602 (SSP-004184AQ) or Exjade® (deferasirox) for Treatment of Chronic Transfusional Iron Overload
Summary
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EudraCT number |
2013-000743-33 |
Trial protocol |
BE GB GR IT |
Global end of trial date |
15 Jul 2014
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Results information
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Results version number |
v1(current) |
This version publication date |
05 Apr 2019
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First version publication date |
05 Apr 2019
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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 |
SPD602-204
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT01927913 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Shire
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Sponsor organisation address |
300 Shire Way, Lexington, United States, MA 02421
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Public contact |
Study Director, Shire, ClinicalTransparency@shire.com
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Scientific contact |
Study Director, Shire, ClinicalTransparency@shire.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? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
15 Jul 2014
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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 |
15 Jul 2014
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Was the trial ended prematurely? |
Yes
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General information about the trial
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Main objective of the trial |
The main objective of this trial was to assess change in liver iron concentration (LIC) from baseline utilizing FerriScan® (R2) magnetic resonance imaging (MRI) in subjects with transfusional iron overload receiving SPD602 (SSP-004184AQ) or deferasirox whose primary diagnosis is beta-thalassemia.
Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. '999999' was entered in Population of Trial Subjects section since '0' could not be entered due to EudraCT system constraints.
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Protection of trial subjects |
This study was conducted in accordance with International Conference on Harmonization (ICH) Good Clinical Practice, the principles of the Declaration of Helsinki, as well as other applicable local ethical and legal requirements.
Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. '999999' was entered in Population of Trial Subjects section since '0' could not be entered due to EudraCT system constraints.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
15 Jul 2014
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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 |
Belgium: 999999
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Worldwide total number of subjects |
999999
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EEA total number of subjects |
999999
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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) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
999999
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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 |
Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. | |||||||||||||||
Period 1
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Period 1 title |
Period 1 (overall period)
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Is this the baseline period? |
Yes | |||||||||||||||
Allocation method |
Randomised - controlled
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Blinding used |
Not blinded | |||||||||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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SPD602 | |||||||||||||||
Arm description |
Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. '99999' was entered since '0' could not be entered due to EudraCT system constraints. | |||||||||||||||
Arm type |
Experimental | |||||||||||||||
Investigational medicinal product name |
SPD602
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Capsule
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Routes of administration |
Oral use
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Dosage and administration details |
16-75 mg/kg/day
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Arm title
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Deferasirox | |||||||||||||||
Arm description |
Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. '99999' was entered since '0' could not be entered due to EudraCT system constraints. | |||||||||||||||
Arm type |
Active comparator | |||||||||||||||
Investigational medicinal product name |
Deferasirox
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Per approved country specific label
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Notes [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same. Justification: Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. '99999' was entered for Number of Subjects for each arm since '0' could not be entered due to EudraCT system constraints. |
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Baseline characteristics reporting groups
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Reporting group title |
SPD602
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Reporting group description |
Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. '99999' was entered since '0' could not be entered due to EudraCT system constraints. | ||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Deferasirox
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Reporting group description |
Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. '99999' was entered since '0' could not be entered due to EudraCT system constraints. | ||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
SPD602
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Reporting group description |
Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. '99999' was entered since '0' could not be entered due to EudraCT system constraints. | ||
Reporting group title |
Deferasirox
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Reporting group description |
Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. '99999' was entered since '0' could not be entered due to EudraCT system constraints. |
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End point title |
Change from baseline in Liver Iron Concentration (LIC) as assessed by FerriScan® R2 at Weeks 12, 24, and 48. [1] | ||||||||||||
End point description |
Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started.
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End point type |
Primary
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End point timeframe |
Baseline to Weeks 12, 24, and 48.
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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: Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. |
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Notes [2] - Study was withdrawn with 0 participants. [3] - Study was withdrawn with 0 participants. |
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No statistical analyses for this end point |
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Adverse events information [1]
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Timeframe for reporting adverse events |
All AEs are collected from the time the informed consent is signed until the defined follow-up period.
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Adverse event reporting additional description |
Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started.
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Assessment type |
Non-systematic | ||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||
Dictionary version |
NA
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Frequency threshold for reporting non-serious adverse events: 5% | |||
Notes [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported. Justification: Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. |
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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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03 Oct 2013 |
Clarified that total daily dose range will be 16-75 mg/kg/day. Modified starting doses for this study from 32, 50, and 75 mg/kg/day to 16 and 20mg/kg BID. Clarified that total daily doses are divided into 2 daily doses. Removed baseline LIC criterion for determination of starting dose; starting dose is determined by TII. Dose adjustments will be made based on LIC, CIC and/or serum ferritin results. Updated EQ-5D-3L to EQ-5D-5L. Updated information on current treatment options to be consistent with newly available data; updated rationale for SPD602 iron overload indication. Updated Inclusion Criteria 4 & 6 and Exclusion Criteria 5, 6 & 9. Clarified that subjects will be stratified by baseline LIC value and historical transfusion requirement after 2-week washout. Updated Rationale for Twice Daily Dosing text. Updated Rationale for the Dosing Algorithm text. Updated acceptable forms of birth control for women of child-bearing potential. Updated in vitro data for drug-drug interaction potential. Revised information regarding suspension or modification of dosing. Added table showing dose titration increments and BID to total daily dose equivalency. Modified dose increments as well as up and down titrations. Indicated that the investigator will be provided with local prescribing information for deferasirox. Updated safety measurements to include serum creatinine and eGFR. Updated renal function assessments to include eGFR. Added laboratory tests related to sensory disturbances as part of the biochemistry panel at select time points and in the event of a neurological adverse event. Specified that Vitamin D is to be assessed as part of biochemistry panel in the event of neurological averse event requiring follow-up. Added LPI to biochemistry panel. Added NTBI and LPI to the clinical pharmacology assessments discussion. Specified blood sample details for neurology-specific
biochemistry testing and updated estimated total mL volume of blood drawn. |
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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. | |||
Study was withdrawn with 0 participants. Sponsor followed EMA's advice and contacted NCAs to enter comment that no participants were recruited. There is still no EudraCT functionality to inform the public that recruitment never started. |