Clinical Trial Results:
An open, prospective trial investigating pharmacokinetics and safety (Part A) of the human normal immunoglobulin for intravenous infusion (IVIG) BT090 and tolerability and safety of escalating infusion rates (Part B) in patients with primary immunodeficiency disease (PID)
Summary
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EudraCT number |
2010-019249-25 |
Trial protocol |
DE HU |
Global end of trial date |
12 Jan 2012
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Results information
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Results version number |
v1(current) |
This version publication date |
15 Sep 2021
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First version publication date |
15 Sep 2021
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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 |
981
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
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WHO universal trial number (UTN) |
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Sponsors
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Sponsor organisation name |
Biotest AG
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Sponsor organisation address |
Landsteinerstr. 5, Dreieich, Germany, 63303
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Public contact |
Corporate Clinical Research and Development, Biotest AG, andrea.wartenberg-demand@biotest.com, Biotest AG, +49 6103801492, 981@biotest.de
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Scientific contact |
Corporate Clinical Research and Development, Biotest AG, andrea.wartenberg-demand@biotest.com, Biotest AG, +49 6103801492, 981@biotest.de
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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 Sep 2012
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
12 Jan 2012
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Global end of trial reached? |
Yes
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Global end of trial date |
12 Jan 2012
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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 |
Investigation of pharmacokinetics (Part A) and tolerability of BT090 at escalating infusion rates (Part B)
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Protection of trial subjects |
none
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Background therapy |
none | ||
Evidence for comparator |
not applicable | ||
Actual start date of recruitment |
15 Nov 2010
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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 |
Germany: 9
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Country: Number of subjects enrolled |
Hungary: 16
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Country: Number of subjects enrolled |
Poland: 5
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Worldwide total number of subjects |
30
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EEA total number of subjects |
30
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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) |
2
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Adolescents (12-17 years) |
5
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Adults (18-64 years) |
23
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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 |
Recruitment period from 15-Nov-2010 (first patient in) until 12-Jan-2012 (last patient out) | ||||||||||||||||
Pre-assignment
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Screening details |
none | ||||||||||||||||
Pre-assignment period milestones
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Number of subjects started |
30 | ||||||||||||||||
Number of subjects completed |
30 | ||||||||||||||||
Period 1
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Period 1 title |
Overall (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||||||
Arms
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Arm title
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Part A + B | ||||||||||||||||
Arm description |
PART A: Pharmacokinetics at 3rd infusion in week 6 or 8 (Cmax, tmax, t1/2el, AUC for serum concentration of IgG and IgG subclasses 1 to 4) and maintenance of IgG trough levels ≥5– 6 g/L. PART B: Tolerability and safety of escalating infusion rates for determination of a maximum tolerated rate. Tolerability was expressed as percentage of total patients being treated at the specified infusion rates on the basis of data received from the 5th and 6th infusions. | ||||||||||||||||
Arm type |
Experimental | ||||||||||||||||
Investigational medicinal product name |
Human normal Immunoglobulin for intravenous use
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Investigational medicinal product code |
BT090
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Other name |
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Pharmaceutical forms |
Infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
In the present trial, the planned monthly dose of BT090 is 200–800 mg/kg BW (2 8 mL/kg BW) administered as intravenous infusions in 3 or 4-week intervals for a treatment period of about 6 months. The dose and dosage intervals must be consistent with pre-trial standard IVIG treatment and are only to be changed if medically indicated
The infusion rates will be increased from 0.3 to 1.4 to 2.0 mL/kg/h for each infusion in each patient at initially 30-minute intervals.
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Baseline characteristics reporting groups
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Reporting group title |
Overall (overall period)
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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 |
All patients who received at least one dose of trial medication (full analysis set).
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Subject analysis set title |
PK Analysis Set
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Subject analysis set type |
Sub-group analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
All patients of the safety set with sufficient data for analysis of pharmacokinetic (PK) parameters.
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Subject analysis set title |
PP Set
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Subject analysis set type |
Per protocol | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
All patients of the safety set without any major protocol violations. Patients with major protocol deviations or incomplete documentation of relevant data or premature termination of the treatment due to reasons that were definitely not related to trial medication will be excluded from the per-protocol analysis.
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End points reporting groups
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Reporting group title |
Part A + B
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Reporting group description |
PART A: Pharmacokinetics at 3rd infusion in week 6 or 8 (Cmax, tmax, t1/2el, AUC for serum concentration of IgG and IgG subclasses 1 to 4) and maintenance of IgG trough levels ≥5– 6 g/L. PART B: Tolerability and safety of escalating infusion rates for determination of a maximum tolerated rate. Tolerability was expressed as percentage of total patients being treated at the specified infusion rates on the basis of data received from the 5th and 6th infusions. | ||
Subject analysis set title |
Safety Set
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
All patients who received at least one dose of trial medication (full analysis set).
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Subject analysis set title |
PK Analysis Set
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Subject analysis set type |
Sub-group analysis | ||
Subject analysis set description |
All patients of the safety set with sufficient data for analysis of pharmacokinetic (PK) parameters.
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Subject analysis set title |
PP Set
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Subject analysis set type |
Per protocol | ||
Subject analysis set description |
All patients of the safety set without any major protocol violations. Patients with major protocol deviations or incomplete documentation of relevant data or premature termination of the treatment due to reasons that were definitely not related to trial medication will be excluded from the per-protocol analysis.
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End point title |
Serum IgG concentration (Cmax) [1] | ||||||||||||
End point description |
Standard Pharmacokinetic Parameter: the maximum observed serum IgG concentration
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End point type |
Primary
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End point timeframe |
At the 3rd infusion (week 6 or 8) standard pharmacokinetic parameters will be determined.
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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: Variables were descriptively summarised. No formal statistical tests were planned or performed. |
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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 |
Recording of AEs commences at the time when the patient is enrolled into the trial (date of signature of the informed consent) until the end of trial visit has been performed.
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Adverse event reporting additional description |
not applicable
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
13.1
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Reporting groups
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Reporting group title |
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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06 Apr 2011 |
SA4 (Protocol Amendment 1) /Prot. Version 2.0: Adaptation of Inclusion criteria/ Section 11.4 |
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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 |