Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43851   clinical trials with a EudraCT protocol, of which   7283   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A randomised, cross-over study to compare quality of life and satisfaction in primary immunodeficient patients treated with subcutaneous injections of Gammanorm® 165 mg/mL administered with two different delivery devices: injections using pump or rapid push.

    Summary
    EudraCT number
    		 2014-003746-27
    Trial protocol
    		 DE   GB  
    Global end of trial date
    11 Dec 2017

    Results information
    Results version number
    		 v1(current)
    This version publication date
    16 Feb 2019
    First version publication date
    16 Feb 2019
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    GAN-06
    Additional study identifiers
    ISRCTN number
    		 ISRCTN55938644
    US NCT number
    		 NCT02503293
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 DRKS: DRKS00008784
    Sponsors
    Sponsor organisation name
    Octapharma Pharmazeutika Produktionsges.m.b.H.
    Sponsor organisation address
    Oberlaaer Strasse 235, Vienna, Austria, 1100
    Public contact
    Clinical Research and Development, Octapharma Pharmazeutika Produktionsges.m.b.H., tatiana.lavrova@octapharma.com
    Scientific contact
    Clinical Research and Development, Octapharma Pharmazeutika Produktionsges.m.b.H., tatiana.lavrova@octapharma.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    27 Nov 2018
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    11 Dec 2017
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To compare satisfaction (LQI questionnaire, factor I: treatment interference) in PID patients receiving subcutaneous injections of Gammanorm 165 mg/mL by delivery device used.
    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 and safety labs.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    07 Jul 2015
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 13
    Country: Number of subjects enrolled
    Germany: 7
    Country: Number of subjects enrolled
    Italy: 5
    Country: Number of subjects enrolled
    Australia: 5
    Worldwide total number of subjects
    30
    EEA total number of subjects
    25
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    27
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Male or female patients suffering from PID and having received subcutaneous injections of immunoglobulin at home using an automatic pump or syringe for at least 1 month at the time of inclusion were eligible for study inclusion.

    Period 1
    Period 1 title
    Overall Trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Gammanorm 165 mg/mL
    Arm description
    		 Each patient received Gammanorm using each of the two studied delivery devices according to the sequence randomly assigned based on a cross-over design in a 1:1 ratio to one of two following sequences: Pump and then syringe, or syringe and then pump.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Gammanorm 165 mg/mL
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    The usual dose was 0.6 mL (100 mg) of Gammanorm 165 mg/mL per kg of body weight per week, which could be administered at several infusion sites. Pump: Initial recommended infusion rate was 15 mL per hour per site. For subsequent infusions, the flow rate could be gradually increased at a rate of 1-2 mL/hour/site to 25 mL/hour/site, as tolerated. The maximum flow rate administered, if tolerated, could have been 100 mL/hour at all sites. Syringe: The usual dose was 0.6 mL (100 mg) of Gammanorm 165 mg/mL per kg of body weight/ week. The weekly dose could have been divided into three injections administered every other day. Maximum infusion rate was set at approximately 1 2 mL/minute. The maximum volume to be infused per injection site was not to exceed 25 mL. If, in general, higher infusion rates and volumes were determined by the routine practice of the site, then higher infusion volumes and higher infusion rates could be administered, if well tolerated by the patient.

    Number of subjects in period 1
    		Gammanorm 165 mg/mL
    Started
    30
    Completed
    28
    Not completed
    2
         Adverse event, non-fatal
    1
         Lost to follow-up
    1

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Overall Trial
    Reporting group description
    		 -

    Reporting group values
    		 Overall Trial Total
    Number of subjects
    		 30 30
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 27 27
        From 65-84 years
    		 3 3
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (full range (min-max))
    		 46.2 (20 to 73) -
    Gender categorical
    Units: Subjects
        Female
    		 15 15
        Male
    		 15 15

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Gammanorm 165 mg/mL
    Reporting group description
    		 Each patient received Gammanorm using each of the two studied delivery devices according to the sequence randomly assigned based on a cross-over design in a 1:1 ratio to one of two following sequences: Pump and then syringe, or syringe and then pump.

    Subject analysis set title
    FAS Population
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Analysis of all randomised patients who have received at least one infusion of immunoglobulin and with at least one baseline evaluation (V1) and one evaluation on treatment (V2 or V3) regarding their treatment satisfaction using the LQI scale.

    Subject analysis set title
    PP Population
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All subjects in the ITT analysis population who completed the trial without significantly violating the inclusion/exclusion criteria or other aspects of the protocol considered to potentially affect the evaluation of the primary endpoint.

    Primary: Lsmean Endpoint Pump Sequence

    		 Close Top of page
    End point title
    		 Lsmean Endpoint Pump Sequence [1]
    End point description
    Assessment of patient satisfaction regarding the treatment delivery device using the LQI I patient satisfaction score (factor I: treatment interference) at the end of the 3-month treatment period with pump.
    End point type
    Primary
    End point timeframe
    at the end of the 3 months treatment period for delivery device pump
    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: One reporting group only, therefore only results of this endpoint (treatment interference) can be documented.
    End point values
    		 FAS Population PP Population
    Number of subjects analysed
    29
    26
    Units: Score
        least squares mean (confidence interval 95%)
    84.32 (79.80 to 89.09)
    83.21 (78.60 to 88.08)
    No statistical analyses for this end point

    Primary: Lsmean Endpoint Syringe Sequence

    		 Close Top of page
    End point title
    		 Lsmean Endpoint Syringe Sequence [2]
    End point description
    Assessment of patient satisfaction regarding the treatment delivery device using the LQI I patient satisfaction score (factor I: treatment interference) at the end of the 3-month treatment period with syringe.
    End point type
    Primary
    End point timeframe
    at the end of the 3 months treatment period for delivery device syringe
    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: One reporting group only, therefore only results of this endpoint (treatment interference) can be documented.
    End point values
    		 FAS Population PP Population
    Number of subjects analysed
    29
    26
    Units: Score
        least squares mean (confidence interval 95%)
    78.85 (74.74 to 83.18)
    78.68 (74.33 to 83.29)
    No statistical analyses for this end point

    Primary: Ratio of Lsmeans Syringe/Pump

    		 Close Top of page
    End point title
    		 Ratio of Lsmeans Syringe/Pump [3]
    End point description
    Ratio of Lsmeans Syringe and Lsmeans Pump.
    End point type
    Primary
    End point timeframe
    at the end of the 3 months treatment period for each delivery device.
    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: Study tested that LQI Factor I (treatment interference) for administration of IMP with syringe is not inferior to pump. Non-inferiority threshold for the ratio LQI Factor I syringe / LQI Factor I pump was set at 0.9. Results were expressed as mean of ratios and two-sided 95% CI. The lower limit of the CI was compared to the non-inferiority threshold of 0.90.n of ratios and two-sided 95% CI. The lower limit of the CI was compared to the non-inferiority threshold of 0.90.
    End point values
    		 FAS Population PP Population
    Number of subjects analysed
    29
    26
    Units: Ratio
        least squares mean (confidence interval 95%)
    93.51 (87.58 to 99.84)
    94.56 (88.53 to 100.99)
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Throughout the whole study from V1 up to V3.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.0
    Reporting groups
    Reporting group title
    Safety Set Total
    Reporting group description
    		 Patients who received at least one dose of IMP.

    Reporting group title
    Safety Set Pump
    Reporting group description
    		 -

    Reporting group title
    Safety Set Syringe
    Reporting group description
    		 -

    Serious adverse events
    		 Safety Set Total Safety Set Pump Safety Set Syringe
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 30 (10.00%)
    1 / 29 (3.45%)
    3 / 30 (10.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Vascular disorders
    Multiple Thromboembolic Event
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 29 (0.00%)
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    C6 nerve impingement
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 29 (3.45%)
    0 / 30 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pain in multiple sites
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 29 (0.00%)
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal bleed
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 29 (0.00%)
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Exacerbation of Asthma
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 29 (3.45%)
    0 / 30 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Sinusitis
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 29 (0.00%)
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 29 (0.00%)
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 29 (0.00%)
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Safety Set Total Safety Set Pump Safety Set Syringe
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    27 / 30 (90.00%)
    24 / 29 (82.76%)
    23 / 30 (76.67%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    2 / 30 (6.67%)
    2 / 29 (6.90%)
    1 / 30 (3.33%)
         occurrences all number
    3
    2
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    6 / 30 (20.00%)
    4 / 29 (13.79%)
    2 / 30 (6.67%)
         occurrences all number
    13
    11
    2
    Dizziness
         subjects affected / exposed
    3 / 30 (10.00%)
    2 / 29 (6.90%)
    2 / 30 (6.67%)
         occurrences all number
    6
    4
    2
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    5 / 30 (16.67%)
    4 / 29 (13.79%)
    3 / 30 (10.00%)
         occurrences all number
    10
    4
    6
    Chills
         subjects affected / exposed
    4 / 30 (13.33%)
    3 / 29 (10.34%)
    2 / 30 (6.67%)
         occurrences all number
    41
    7
    34
    Fatigue
         subjects affected / exposed
    3 / 30 (10.00%)
    3 / 29 (10.34%)
    1 / 30 (3.33%)
         occurrences all number
    9
    8
    1
    Feeling Cold
         subjects affected / exposed
    2 / 30 (6.67%)
    2 / 29 (6.90%)
    1 / 30 (3.33%)
         occurrences all number
    47
    8
    39
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    5 / 30 (16.67%)
    4 / 29 (13.79%)
    2 / 30 (6.67%)
         occurrences all number
    15
    10
    5
    Abdominal discomfort
         subjects affected / exposed
    2 / 30 (6.67%)
    1 / 29 (3.45%)
    1 / 30 (3.33%)
         occurrences all number
    2
    1
    1
    Abdominal pain
         subjects affected / exposed
    2 / 30 (6.67%)
    2 / 29 (6.90%)
    0 / 30 (0.00%)
         occurrences all number
    3
    3
    0
    Abdominal pain upper
         subjects affected / exposed
    2 / 30 (6.67%)
    1 / 29 (3.45%)
    1 / 30 (3.33%)
         occurrences all number
    2
    1
    1
    Diarrhoea
         subjects affected / exposed
    2 / 30 (6.67%)
    2 / 29 (6.90%)
    0 / 30 (0.00%)
         occurrences all number
    2
    2
    0
    Vomiting
         subjects affected / exposed
    2 / 30 (6.67%)
    1 / 29 (3.45%)
    1 / 30 (3.33%)
         occurrences all number
    2
    1
    1
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    5 / 30 (16.67%)
    5 / 29 (17.24%)
    1 / 30 (3.33%)
         occurrences all number
    7
    6
    1
    Cough
         subjects affected / exposed
    4 / 30 (13.33%)
    5 / 29 (17.24%)
    2 / 30 (6.67%)
         occurrences all number
    7
    6
    2
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    4 / 30 (13.33%)
    2 / 29 (6.90%)
    2 / 30 (6.67%)
         occurrences all number
    12
    2
    10
    Arthralgia
         subjects affected / exposed
    3 / 30 (10.00%)
    1 / 29 (3.45%)
    2 / 30 (6.67%)
         occurrences all number
    13
    1
    12
    Myalgia
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 29 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    16
    0
    16
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    7 / 30 (23.33%)
    7 / 29 (24.14%)
    2 / 30 (6.67%)
         occurrences all number
    11
    9
    2
    Nasopharyngitis
         subjects affected / exposed
    7 / 30 (23.33%)
    3 / 29 (10.34%)
    4 / 30 (13.33%)
         occurrences all number
    7
    3
    4
    Lower respiratory tract infection
         subjects affected / exposed
    4 / 30 (13.33%)
    2 / 29 (6.90%)
    3 / 30 (10.00%)
         occurrences all number
    6
    3
    3
    Sinusitis
         subjects affected / exposed
    4 / 30 (13.33%)
    2 / 29 (6.90%)
    4 / 30 (13.33%)
         occurrences all number
    8
    2
    6
    Oral candidiasis
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 29 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    3
    0
    3
    Pharyngitis
         subjects affected / exposed
    2 / 30 (6.67%)
    2 / 29 (6.90%)
    1 / 30 (3.33%)
         occurrences all number
    3
    2
    1
    Rhinitis
         subjects affected / exposed
    2 / 30 (6.67%)
    1 / 29 (3.45%)
    1 / 30 (3.33%)
         occurrences all number
    2
    1
    1
    Urinary tract infection
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 29 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    2
    0
    2

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    09 Mar 2015
    Amendment 1 - Implementation of the requested changes regarding biostatistics by the authorities in Germany and clarifications to the protocol
    02 Jun 2015
    Amendment 3: - Higher infusion rates and higher volumes may be administered if determined by the routine practice of the site. - Unscheduled Visits were added.
    07 Dec 2015
    Amendment 4: - Clarification of Adverse Events documentation - Addition of study sites in Australia
    10 Oct 2016
    Amendment 10: - Prolongation of study duration - maximum number of patients enrolled increased from 30 to 40

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat Apr 20 09:37:17 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA