Clinical Trial Results:
An international, multicentre, efficacy and safety study of I10E in initial and maintenance treatment of patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy.
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Summary
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EudraCT number |
2013-005557-73 |
Trial protocol |
GB ES IT FR DE |
Global end of trial date |
29 Sep 2017
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Results information
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Results version number |
v1(current) |
This version publication date |
15 Jan 2020
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First version publication date |
15 Jan 2020
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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 |
I10E-1302
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Additional study identifiers
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ISRCTN number |
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US NCT number |
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WHO universal trial number (UTN) |
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Sponsors
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Sponsor organisation name |
LFB Biotechnologies
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Sponsor organisation address |
3 Avenue des Tropiques, COURTABOEUF, France, 91930
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Public contact |
Global Clinical Development Leader , LFB BIOTECHNOLOGIES, +33 1 69 82 70 10,
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Scientific contact |
Global Clinical Development Leader , LFB BIOTECHNOLOGIES, +33 1 69 82 70 10,
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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 |
18 Dec 2019
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
29 Sep 2017
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Global end of trial reached? |
Yes
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Global end of trial date |
29 Sep 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 primary objective of this study is to assess the efficacy of I10E in improving the disability of patients with CIDP.
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Protection of trial subjects |
This study was conducted in compliance with good clinical practice (GCP) as described in the
International Conference on Harmonisation (ICH) document “Guidance for Industry-E6 Good Clinical
Practice: Consolidated Guidance” dated April 1996. These practices were consistent with the principles
stated in the Declaration of Helsinki (October 2013 revised version). All other applicable regulations
were followed.
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Background therapy |
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Evidence for comparator |
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Actual start date of recruitment |
24 Feb 2015
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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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 |
Tunisia: 8
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Country: Number of subjects enrolled |
Turkey: 3
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Country: Number of subjects enrolled |
Poland: 8
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Country: Number of subjects enrolled |
Spain: 6
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Country: Number of subjects enrolled |
United Kingdom: 1
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Country: Number of subjects enrolled |
France: 2
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Country: Number of subjects enrolled |
Italy: 16
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Worldwide total number of subjects |
44
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EEA total number of subjects |
33
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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) |
32
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From 65 to 84 years |
12
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85 years and over |
0
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Recruitment
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Recruitment details |
Between 24 Feb 2015 and 22 Mar 2017, 59 subjects from 23 sites signed an informed consent. | ||||||||||||||||
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Pre-assignment
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Screening details |
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Pre-assignment period milestones
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Number of subjects started |
59 [1] | ||||||||||||||||
Number of subjects completed |
43 | ||||||||||||||||
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Pre-assignment subject non-completion reasons
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Reason: Number of subjects |
Consent withdrawn by subject: 1 | ||||||||||||||||
Reason: Number of subjects |
Screening failure: 15 | ||||||||||||||||
| Notes [1] - The number of subjects reported to have started the pre-assignment period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same. Justification: 59 subjects signed an informed consent in the pre-assigment period. Among them, 18 subjects were considered as a screening failure including 3 subjects re-screened and finally enrolled. Of 44 enrolled subjects, one subject was discontinued (consent withdrawn by subject) from the study before the first administration of study drug. |
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Period 1
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Period 1 title |
Treatment period (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 | ||||||||||||||||
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Arms
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Arm title
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Single arm | ||||||||||||||||
Arm description |
This study included patients never previously treated with IgG and patients already treated with IgG but in clinical relapse following IgG therapy discontinuation. | ||||||||||||||||
Arm type |
Experimental | ||||||||||||||||
Investigational medicinal product name |
IQYMUNE
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Investigational medicinal product code |
I10E
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Other name |
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Pharmaceutical forms |
Solution for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
I10E (study drug) was administered at 2 g/kg over 2 to 5 days for the first course, then at 1 g/kg over 1 to 2 days for 7 additional courses at 3-week intervals.
Study drug was administered intravenously, with an infusion pump (B-Braun Infusomat Space).
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| Notes [2] - 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: Of 44 enrolled subjects, one patient was discontinued (consent withdrawn by subject) from the study before the first administration of study drug. |
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Baseline characteristics reporting groups
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Reporting group title |
Treatment period
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Reporting group description |
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Subject analysis sets
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Subject analysis set title |
Total Treated Set
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Subject analysis set type |
Safety analysis | ||||||||||||||||||||||||||||||||||||
Subject analysis set description |
All subjects who received at least one infusion of I10E
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Subject analysis set title |
Full Analysis Set
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Subject analysis set type |
Full analysis | ||||||||||||||||||||||||||||||||||||
Subject analysis set description |
All TTS patients having an available assessment of the primary efficacy criteria.
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End points reporting groups
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Reporting group title |
Single arm
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Reporting group description |
This study included patients never previously treated with IgG and patients already treated with IgG but in clinical relapse following IgG therapy discontinuation. | ||
Subject analysis set title |
Total Treated Set
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
All subjects who received at least one infusion of I10E
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Subject analysis set title |
Full Analysis Set
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Subject analysis set type |
Full analysis | ||
Subject analysis set description |
All TTS patients having an available assessment of the primary efficacy criteria.
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End point title |
Responder Rate [1] | ||||||
End point description |
The primary efficacy endpoint was the responder rate at End of Study (EoS). Responders were defined as patients with a decrease ≥1 point in the adjusted INCAT disability score compared to baseline.
If a patient was treated with a not-allowed treatment during the study period, then all adjusted INCAT disability score measured after the intake of these not-allowed treatments were censored.
If the score at EoS visit was missing, then the Last Observation Carried Forward (LOCF) approach was applied to replace this missing value.
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End point type |
Primary
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End point timeframe |
During treatment period the adjusted INCAT disability score was evaluated at week 3, week 6, week 9, week 12, week 15, week 18, week 21, week 24 (End-of-Study).
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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: For this single-arm study, the response rate was tested against the historical response rate of 33.3% with a 1-sided Clopper-Pearson exact test at the nominal level of significance of 2.5% on the Full Analysis Set. The null and alternative hypotheses were as follows: H0: pI10E <= 33.3% H1: pI10E > 33.3% The estimate of the response rate was 76.2% (Exact 2-sided 95% Clopper-Pearson CI[60.5%;87.9%]). The p-value of the 1-sided Clopper-Pearson exact test was < 0.0001. |
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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 |
throughout the study
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Assessment type |
Non-systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
17.1
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Reporting groups
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Reporting group title |
Total treated Set
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Reporting group description |
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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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10 Mar 2016 |
- Modification of some exclusion criteria
- Removal of routine free bilirubin testing
- Additional renal safety follow-up
- Description of the patient population
- Rules for follow-up of adverse events
- Timing of safety monitoring (vital signs)
- Update of IMP storage conditions
This Amendment led to protocol version 5.0 dated 06 January 2016 |
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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 | |||
