Clinical Trial Results:
Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID)
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Summary
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EudraCT number |
2007-002611-27 |
Trial protocol |
DE FR GB |
Global end of trial date |
30 Sep 2010
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Results information
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Results version number |
v1(current) |
This version publication date |
31 Dec 2016
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First version publication date |
31 Dec 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 |
GAM10-03
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Additional study identifiers
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ISRCTN number |
ISRCTN63491981 | ||
US NCT number |
NCT00811174 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Octapharma AG
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Sponsor organisation address |
Seidenstrasse 2, Lachen, Switzerland, CH-8853
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Public contact |
Clinical Research Department, Octapharma Pharmazeutika Prod.Ges.m.b.H.
, 0043 (1)61032-0,
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Scientific contact |
Clinical Research Department, Octapharma Pharmazeutika Prod.Ges.m.b.H.
, 0043 (1)61032-0,
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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 |
12 Sep 2012
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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 |
30 Sep 2010
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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 primary objective of the study is to investigate the safety of Octagam® 10% in
replacement therapy in PID and to compare the pharmacokinetic profile of Octagam® 10%with that of the previously used commercial Octagam® 5% (Pharmacokinetic sub-study).
Safety outcome parameters will be:
• Occurrence of adverse events.
• Short term tolerance parameters including vital signs (blood pressure, heart rate,
temperature, respiratory rate).
• Laboratory parameters (hematology, clinical chemistry, direct Coombs’test,
urinalysis) and tests for viral safety.
Pharmacokinetic outcome parameters will be the parameter Cmax, Cmin, t ½, Tmax, AUC, volume of distribution, and incremental recovery of serum total IgG; of IgG subclasses (IgG1, IgG2, IgG3, IgG4); of specific antibodies against Haemophilus influenzae, Streptococcus pneumoniae (types 4, 6B, 9V, 14, 18C, 19F, 23F), CMV, VZV, tetanus, measles; and of glucose and maltose.
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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 Declaration of Helsinki. Inclusion and exclusion criteria were carefully defined in order to protect subjects from contraindications, interactions with other medication and risk factors associated with the investigational medicinal product. Throughout the study safety was assessed, such as occurrence of AEs, safety labs, vital signs and physical examinations.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
31 Mar 2009
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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 |
Poland: 2
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Country: Number of subjects enrolled |
Germany: 3
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Worldwide total number of subjects |
5
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EEA total number of subjects |
5
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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) |
2
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Adults (18-64 years) |
3
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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 was planned to be performed in patients with PID requiring antibody replacement therapy and who had received Octagam 5% replacement therapy at a steady dose and schedule for at least six infusions up to study entry | ||||||||||
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Pre-assignment
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Screening details |
The study was planned to be performed in patients with PID requiring antibody replacement therapy and who had received Octagam 5% replacement therapy at a steady dose and schedule for at least six infusions up to study entry.Screening was to be performed between the end of the last pre-study infusion and the first infusion in the study | ||||||||||
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Period 1
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Period 1 title |
Enrolled Patients (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 | ||||||||||
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Arms
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Arm title
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Octagam 10% | ||||||||||
Arm description |
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Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
Octagam 10% ,Human immunoglobulin
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Investigational medicinal product code |
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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 |
Depending on the regular treatment intervals (every 3 or 4 weeks) the patient was to receive 17 or 13 infusions of Octagam 10% in the course of this study. The dose administered per kg body weight and the treatment intervals were to remain the same throughout the study, as long as trough levels of serum IgG were maintained above 5 g/L.
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Baseline characteristics reporting groups
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Reporting group title |
Enrolled Patients (Overall Study)
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Octagam 10%
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Reporting group description |
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End point title |
Serum IgG trough levels before each administration (pre-next dose) [1] | ||||||||
End point description |
Owing to the limited data available (only 5 patients enrolled, of whom 2 completed the study and 3 ended treatment prematurely), efficacy and PK analyses were not performed.
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End point type |
Primary
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End point timeframe |
Screening visit until the last study visit
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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 study was terminated prematurely. Owing to the limited data available (only 5 patients enrolled, of whom 2 completed the study and 3 ended treatment prematurely), efficacy and PK analyses were not performed. |
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| Notes [2] - Because of the premature termination no analyses was done |
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End point title |
IgG-profile pharmacokinetics determined at the last but one (or last) infusion [3] | ||||||||
End point description |
Owing to the limited data available (only 5 patients enrolled, of whom 2 completed the study and 3 ended treatment prematurely), efficacy and PK analyses were not performed.
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End point type |
Primary
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End point timeframe |
From screening visit to last visit of the study
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| Notes [3] - 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 study was terminated prematurely. Owing to the limited data available (only 5 patients enrolled, of whom 2 completed the study and 3 ended treatment prematurely), efficacy and PK analyses were not performed. |
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| Notes [4] - Because of the premature termination no analyses was done |
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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 were assessed throughout the whole study from screening visit to the last visit 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 |
11.0
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Reporting groups
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Reporting group title |
Octagam 10%
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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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13 May 2009 |
• Inclusion criterion of primary immunodeficiency requiring immunoglobulin replacement therapy must be associated with hypo- or agammaglobulinemia
• Consistent dose to be infused is widened to 200 – 800 mg/kg BW
• Update of drug safety section according to new drug safety SOP
• Change of study duration
• Terminology “anonymity/anonymous/anonymised” is replaced by appropriate wording
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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 | |||||||
