Clinical Trial Results:
Prospective, Open-label, Non-controlled, Multicenter, Phase III Clinical Study to Evaluate the Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NEWGAM) in Primary Immune Thrombocytopenia
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Summary
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EudraCT number |
2009-014589-24 |
Trial protocol |
DE CZ BG FR |
Global end of trial date |
22 Jul 2013
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Results information
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Results version number |
v1(current) |
This version publication date |
28 Jul 2016
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First version publication date |
28 Jul 2016
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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 |
NGAM-02
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT01349790 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Octapharma Pharmazeutika Produktionsgesellschaft mbH
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Sponsor organisation address |
Oberlaaer Strasse 235 A-1100 Vienna Austria , Vienna , Austria, A-1100
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Public contact |
Clinical Research and Development, Octapharma Pharmazeutika Produktionsgesellschaft mbH, 0043 161032, clinical.department@octapharma.com
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Scientific contact |
Clinical Research and Development, Octapharma Pharmazeutika Produktionsgesellschaft mbH, 0043 161032, clinical.department@octapharma.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 |
05 Mar 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 |
22 Jul 2013
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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 |
To assess the efficacy of NewGam in correcting the platelet count.
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Protection of trial subjects |
This trial was conducted in accordance to the principles of GCP, ensuring that the rights, safety and well-being of patients are protected and in consistency with the the Declaration of Helsinki.
Inclusion and exclusion criteria were carefully defined in order to protect subjects from contraindications, interactions with other medication and safety factors associated with the investigational medicinal product.
Throughout the study safety was assessed, such as occurrence of AEs, labvalues, vital signs and physical examinations.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
27 Oct 2011
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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 |
Poland: 4
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Country: Number of subjects enrolled |
Bulgaria: 3
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Country: Number of subjects enrolled |
Czech Republic: 8
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Country: Number of subjects enrolled |
Germany: 3
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Country: Number of subjects enrolled |
Romania: 3
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Country: Number of subjects enrolled |
Russian Federation: 8
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Country: Number of subjects enrolled |
Ukraine: 7
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Country: Number of subjects enrolled |
India: 4
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Worldwide total number of subjects |
40
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EEA total number of subjects |
21
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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) |
38
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From 65 to 84 years |
2
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85 years and over |
0
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Recruitment
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Recruitment details |
- | ||||||||||||||||||
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Pre-assignment
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Screening details |
40 Patients were enrolled at 20 centres, located in Germany, Czech Republic, Russia, Bulgaria, India, Poland, Romania and Ukraine. | ||||||||||||||||||
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Period 1
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Period 1 title |
overall trial (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||||||||
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Arms
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Arm title
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NewGam | ||||||||||||||||||
Arm description |
total dose of 2g/kg NewGam, human normal immunoglobulin 10%, for treatment in primary immune thrombocytopenia on 2 consecutive days | ||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||
Investigational medicinal product name |
NewGam, human normal immunoglobulin 10%
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for injection/infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Infusion of 1 g/kg per day on 2 consecutive days.
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Baseline characteristics reporting groups
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Reporting group title |
overall trial
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Reporting group description |
NewGam 10% | ||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
Full Analysis Set
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Subject analysis set type |
Intention-to-treat | ||||||||||||||||||||||||||||||||||||
Subject analysis set description |
consists of all patients in the Safety Set who satisfy all major eligibility criteria and for whom at least 1 post-baseline measurement of platelet concentration data is available. This is the set of eligible patients with treatment effects measured, according to the intention-to-treat (ITT) principle.
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Subject analysis set title |
First Per Protocol (PP1) Set
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Subject analysis set type |
Per protocol | ||||||||||||||||||||||||||||||||||||
Subject analysis set description |
All patients of the FA set excluding those who had major protocol violations before the primary efficacy endpoint (Day 8) was reached and which may have had an impact on the evaluation of the primary endpoint
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End points reporting groups
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Reporting group title |
NewGam
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Reporting group description |
total dose of 2g/kg NewGam, human normal immunoglobulin 10%, for treatment in primary immune thrombocytopenia on 2 consecutive days | ||
Subject analysis set title |
Full Analysis Set
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Subject analysis set type |
Intention-to-treat | ||
Subject analysis set description |
consists of all patients in the Safety Set who satisfy all major eligibility criteria and for whom at least 1 post-baseline measurement of platelet concentration data is available. This is the set of eligible patients with treatment effects measured, according to the intention-to-treat (ITT) principle.
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Subject analysis set title |
First Per Protocol (PP1) Set
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Subject analysis set type |
Per protocol | ||
Subject analysis set description |
All patients of the FA set excluding those who had major protocol violations before the primary efficacy endpoint (Day 8) was reached and which may have had an impact on the evaluation of the primary endpoint
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End point title |
Response Rate [1] | ||||||||||||
End point description |
The primary endpoint of this study was the response rate, i.e., the proportion of patients with an elevation of platelet count to greater equal 50x10^9/L within 7 days of the first infusion, i.e., by study Day 8 (at least once prior to Day 9). The evaluation of the primary objective was performed for the FA set (ITT analysis) and for the PP1 set (PP analysis).
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End point type |
Primary
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End point timeframe |
Baseline, Day 2 to Day 8.
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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: The analysis of the primary objective aimed at demonstrating that the response rate after administration of NewGam (p), defined as the proportion of patients with an elevation of platelet count to gretater equal 50x10^9/L within 7 days after the first infusion, was above a pre-defined value of 0.60. This threshold was obtained from a historical control value of p0=0.75 and a region of indifference ofδ=0.15. The hypothesis (H0: p ≤ p0−δ) was tested at a 1-sided significance level of α =0.025. |
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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 |
AE were evaluated at each visit until day 63 of the study
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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 |
14
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Reporting groups
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Reporting group title |
All patients exposed to treatment ( Safety 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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22 Nov 2011 |
Amendment #2:
The inclusion criterion for age was narrowed to at least 18 years and no older than 65 years old (was 80 years)
Two new exclusion criteria added:
• Patients with BMI greater than or equal to 30 kg/m2
• Patients with risk factors for TEE in which the risks outweigh the potential benefit of NewGam treatment
Medically measureable parameters leading to an early termination of individual patients were implemented.
The concomitant medications and AEs were to be monitored until Day 63 instead of until Day 22.
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14 Jun 2012 |
Amendment #3:
• The exclusion criterion regarding prior rituximab therapy was extended to 4 months from 3 months.
• A second confirmatory assessment was included.
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Interruptions (globally) |
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| Were there any global interruptions to the trial? Yes | |||||||
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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 | |||||||
