Clinical Trial Results:
“CLINICAL STUDY TO EVALUATE THE PHARMACOKINETICS, SAFETY AND EFFICACY OF OCTAGAM 5% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES”
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Summary
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EudraCT number |
2012-000792-16 |
Trial protocol |
HU CZ DE |
Global end of trial date |
12 Mar 2013
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Results information
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Results version number |
v1(current) |
This version publication date |
22 Jul 2016
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First version publication date |
22 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 |
GAMr-29
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
- | ||
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 Produktionsgesellschaft, +43 1 61032 1295, clinical.department@octapharma.com
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Scientific contact |
Clinical Research Department, Octapharma Pharmazeutika Produktionsgesellschaft, +43 1 61032 1295, 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 |
03 Feb 2014
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
12 Mar 2013
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Global end of trial reached? |
Yes
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Global end of trial date |
12 Mar 2013
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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 |
To determine the pharmacokinetic (PK) profile of Octagam 5% at steady state on standard prophylactic treatment of Primary Immunodeficiency Disorders (PID).
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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 safety factors associated with the investigational medicinal product. Short term tolerance parameters (blood pressure, heart rate, temperature, respiratory rate) and safety laboratory parameters (haematology, clinical chemistry, and urinalysis) were evaluated. Relevant drug safety information (AEs and SAEs) was collected according a safety reporting plan.
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Background therapy |
NA | ||
Evidence for comparator |
NA | ||
Actual start date of recruitment |
04 Jul 2012
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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: 7
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Country: Number of subjects enrolled |
Czech Republic: 8
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Country: Number of subjects enrolled |
Germany: 1
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Country: Number of subjects enrolled |
Hungary: 11
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Worldwide total number of subjects |
27
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EEA total number of subjects |
27
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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) |
24
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From 65 to 84 years |
3
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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 start enrolment during the 2nd quarter of 2012 with an overall study duration of 6 month per patient, so study was completed by 2nd quarter of 2014. | ||||||||||||
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Pre-assignment
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Screening details |
screening was performed between the end of the last pre-study infusion and the first infusion of this study, i.e., at the time the patient´s next treatment with IVIG was due. All results necessary to check the patient´s eligibility had to be available before infusion. | ||||||||||||
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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 | ||||||||||||
Blinding implementation details |
NA
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Arms
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Arm title
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Octagam 5% - 4/3-week treatment schedule | ||||||||||||
Arm description |
Octagam 5%, human normal immunoglobulin 5%, solvent/detergent-treated solution for IV infusion. The dose infused was 200-800 mg/kg body weight every 21 (+/- 3) or 28 (+/-3) days (in Germany and Hungary: "approximately 200-800 mg/kg), with individual doses and intervals being dependent on the patient´s previous IVIG dose and interval before entry into the study. All infusions started with an infusion rate of octagam 5% of 1 mL/kg/h (50 mg/kg/h) for the first 30 minutes; if tolerated, the rate of administration could gradually be increased to a maximum of 5 mL/kg/h (250 mg/kg/h) for the remainder of the infusion. | ||||||||||||
Arm type |
Experimental | ||||||||||||
Investigational medicinal product name |
Octagam 5%, human normal immunoglobulin 5%
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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 |
The dose infused was 200–800 mg/kg body weight every 21 (±3) or 28 (±3) days, (in Germany and Hungary: ‘approximately 200–800 mg/kg body weight every 21 [±3] or 28 [±3] days’), with individual doses and intervals being dependent on the patient’s previous IVIG dose and interval before entry into the study.
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Baseline characteristics reporting groups
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Reporting group title |
Overall trial
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Reporting group description |
- | |||||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
Safety Set
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Subject analysis set type |
Safety analysis | |||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
The safety set comprised all patients who received at least 1 dose of study medication; it was the set of patients exposed to treatment.
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Subject analysis set title |
Completed patients
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Subject analysis set type |
Per protocol | |||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
The per-protocol (PP) set comprised all patients of the FA set excluding those with major protocol violations which may have had an impact on the analysis of efficacy. This was the set of patients who participated in the study as intended and for whom efficacy could be evaluated as planned, even if the PK profile was missing or could not be used for other reasons.
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End points reporting groups
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Reporting group title |
Octagam 5% - 4/3-week treatment schedule
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Reporting group description |
Octagam 5%, human normal immunoglobulin 5%, solvent/detergent-treated solution for IV infusion. The dose infused was 200-800 mg/kg body weight every 21 (+/- 3) or 28 (+/-3) days (in Germany and Hungary: "approximately 200-800 mg/kg), with individual doses and intervals being dependent on the patient´s previous IVIG dose and interval before entry into the study. All infusions started with an infusion rate of octagam 5% of 1 mL/kg/h (50 mg/kg/h) for the first 30 minutes; if tolerated, the rate of administration could gradually be increased to a maximum of 5 mL/kg/h (250 mg/kg/h) for the remainder of the infusion. | ||
Subject analysis set title |
Safety Set
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
The safety set comprised all patients who received at least 1 dose of study medication; it was the set of patients exposed to treatment.
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Subject analysis set title |
Completed patients
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Subject analysis set type |
Per protocol | ||
Subject analysis set description |
The per-protocol (PP) set comprised all patients of the FA set excluding those with major protocol violations which may have had an impact on the analysis of efficacy. This was the set of patients who participated in the study as intended and for whom efficacy could be evaluated as planned, even if the PK profile was missing or could not be used for other reasons.
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End point title |
IgG Cmax [1] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
The total study duration (including screening period) per patient was approximately 6 months.
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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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG1 Cmax [2] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
The total study duration (including screening period) per patient was approx. 6 months
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| Notes [2] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG2 Cmax [3] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG3 Cmax [4] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [4] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Cmin [5] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screeing period) per patient was approx. 6 months.
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| Notes [5] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG1 Cmin [6] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [6] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG2 Cmin [7] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [7] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG3 Cmin [8] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [8] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG4 Cmax [9] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [9] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG4 Cmin [10] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [10] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG AUC0-last [11] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [11] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG1 AUC0-last [12] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [12] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG2 AUC0-last [13] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [13] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG3 AUC0-last [14] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [14] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG4 AUC0-last [15] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [15] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG t1/2 [16] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [16] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG1 t1/2 [17] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [17] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG2 t1/2 [18] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [18] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG3 t1/2 [19] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [19] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG4 t1/2 [20] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [20] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Vd [21] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [21] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG1 Vd [22] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [22] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG2 Vd [23] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [23] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG3 Vd [24] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [24] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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| No statistical analyses for this end point | |||||||||
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End point title |
IgG Subclass IGG4 Vd [25] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
the total study duration (including screening period) per patient was approx. 6 months.
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| Notes [25] - 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: Derived PK parameter; no statistical analysis or comparison performed (single-arm study) |
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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 |
24 hours SAE reporting adverse events
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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 |
12.0
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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? No | |||
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 | |||
