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    Clinical Trial Results:
    Safety and Efficacy of abatacept (s.c.) in patients with CTLA4 insufficiency or LRBA deficiency

    Summary
    EudraCT number
    		 2019-000972-40
    Trial protocol
    		 DE  
    Global end of trial date
    28 Jun 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    02 Jan 2025
    First version publication date
    02 Jan 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    IM101-774
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 DRKS: DRKS00017736
    Sponsors
    Sponsor organisation name
    Medical Center - University of Freiburg
    Sponsor organisation address
    Breisacher Str. 115, Freiburg, Germany, 79106
    Public contact
    Coordinating Investigator: Bodo Grimbacher, Medical Center - University of Freiburg, +49 761270-77731, bodo.grimbacher@uniklinik-freiburg.de
    Scientific contact
    Coordinating Investigator Bodo Grimbacher, Medical Center - University of Freiburg, +49 761270-77731, bodo.grimbacher@uniklinik-freiburg.de
    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
    08 Oct 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    28 Jun 2023
    Global end of trial reached?
    Yes
    Global end of trial date
    28 Jun 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    to assess the safety of abatacept for patients with cytotoxic T-lymphocyte-associated protein 4 (CTLA4) insufficiency or lipopolysaccharide-responsive and beige-like anchor protein (LRBA) deficiency
    Protection of trial subjects
    The investigator was responsible for ensuring that the study was performed in accordance with the ethical principles of the Declaration of Helsinki as well as with the national laws and guidelines for the clinical testing of drugs. Before enrolment in the clinical trial, the patient was informed that participation in the clinical trial is voluntary and that he/she may withdraw from the clinical trial at any time without having to give reasons and without penalty or loss of benefits to which the patient is otherwise entitled.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    09 Jul 2020
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Germany: 24
    Worldwide total number of subjects
    24
    EEA total number of subjects
    24
    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)
    23
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 -

    Pre-assignment period milestones
    Number of subjects started
    		 24
    Number of subjects completed
    		 20

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 Not meeting inclusion criteria: 3
    Reason: Number of subjects
    		 Other reasons: 1
    Period 1
    Period 1 title
    Overall (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    ABACHAI is a phase IIa prospective, non-randomized, open-label, single arm multi-center trial.

    Arms
    Arm title
    		 Abatacept
    Arm description
    		 All patients were treated with abatacept 125 mg once a week as a subcutaneous injection.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Abatacept
    Investigational medicinal product code
    Other name
    Orencia
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Solution for injection
    Dosage and administration details
    Strength: 125 mg/1 ml syringe Dose: 1x 125 mg/1 ml syringe weekly

    Number of subjects in period 1 [1]
    		Abatacept
    Started
    20
    Completed
    20
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: Overall, 24 patients gave their informed consent for study participation. From these, 4 patients were screening failures, i.e. did not met the inclusion/exclusion criteria and were not treated with the study medication. For the other 20 patients, treatment with abatacept as part of the study was approved. Of those, 16 patients completed the study per protocol.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall
    Reporting group description
    		 -

    Reporting group values
    		 Overall Total
    Number of subjects
    		 20 20
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 19 19
        From 65-84 years
    		 1 1
    Age continuous
    Units: years
        median (full range (min-max))
    		 37 (19 to 70) -
    Gender categorical
    Units: Subjects
        Female
    		 14 14
        Male
    		 6 6
    Subject analysis sets

    Subject analysis set title
    Full analysis set (FAS)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The full analysis set (FAS) includes all 20 patients who were treated with at least one dose of abatacept

    Subject analysis sets values
    		 Full analysis set (FAS)
    Number of subjects
    20
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    19
        From 65-84 years
    1
    Age continuous
    Units: years
        median (full range (min-max))
    37 (19 to 70)
    Gender categorical
    Units: Subjects
        Female
    14
        Male
    6

    End points

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    End points reporting groups
    Reporting group title
    Abatacept
    Reporting group description
    		 All patients were treated with abatacept 125 mg once a week as a subcutaneous injection.

    Subject analysis set title
    Full analysis set (FAS)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The full analysis set (FAS) includes all 20 patients who were treated with at least one dose of abatacept

    Primary: Number of episodes of failed infection control under therapy with abatacept during the trial period of one year

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    End point title
    		 Number of episodes of failed infection control under therapy with abatacept during the trial period of one year [1]
    End point description
    Number of episodes of failed infection control under therapy with abatacept during the trial period of one year. An episode of failed infection control was defined as a severe infection, defined as a. Infections requiring hospitalization, OR b. Infectious requiring intravenous antibiotic, anti-fungal or anti-viral treatment, OR/AND c. EBV reactivation (≥ 5.000 IU/ml) or CMV viral load ≥ 1.000 IU/ml.
    End point type
    Primary
    End point timeframe
    During the trial period of one year.
    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: Single arm trial. The number of episodes of failed infection control during the trial period of one year is calculated as an annual incidence rate, and is presented with a two-sided 95% CI.
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20
    Units: Number of episodes
        Number of severe infections during trial period
    5
    No statistical analyses for this end point

    Secondary: Number of severe infections

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    End point title
    		 Number of severe infections
    End point description
    Defined as i.e. requiring hospitalization, or an infection requiring i.v. antibiotic, i.v. anti-fungal, or i.v. anti-viral treatment.
    End point type
    Secondary
    End point timeframe
    the year preceding 1st abatacept application
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20
    Units: Number of severe infections
    5
    No statistical analyses for this end point

    Secondary: Number of episodes of failed infection control exceeding 3 months under therapy

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    End point title
    		 Number of episodes of failed infection control exceeding 3 months under therapy
    End point description
    Number of episodes of failed infection control exceeding 3 months under therapy with abatacept during the trial period of one year. These were as defined above (primary endpoint), with three exceptions: o primary viral infections which were controlled within 0 to 3 months. o hospitalizations which were conducted solely for preventive reasons o i.v. antibiotic, or i.v. anti-fungal, or i.v. anti-viral treatments which were conducted solely for preventive reasons
    End point type
    Secondary
    End point timeframe
    Exceeding 3 months under therapy with abatacept during the trial period of one year.
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20
    Units: Number of episodes
    0
    No statistical analyses for this end point

    Secondary: Characterization of severe infections

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    End point title
    		 Characterization of severe infections
    End point description
    Characterization (incl. type of pathogen and involved organ system) of severe infections (defined as: an infection requiring hospitalization OR an infection requiring i.v. antibiotic, or i.v. anti-fungal, or i.v. anti-viral treatment).
    End point type
    Secondary
    End point timeframe
    During abatacept trial treatment period.
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20
    Units: Characterization
        SARS-CoV-2 infection
    3
        Exacerbated COPD due to infection, DD Covid 19
    1
        Exacerbation of COPD due to infection
    1
    No statistical analyses for this end point

    Secondary: Event free Survival

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    End point title
    		 Event free Survival
    End point description
    Results of the Kaplan-Meier analysis
    End point type
    Secondary
    End point timeframe
    Calculated as time from start of treatment with abatacept as trial medication until death from any cause or treatment failure (premature termination of abatacept treatment for any reason).
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20
    Units: Rates
    number (confidence interval 95%)
        Month 3
    100 (100 to 100)
        Month 6
    95 (69.5 to 99.3)
        Month 12
    80 (55.1 to 92)
        Month 15
    80 (55.1 to 92)
    No statistical analyses for this end point

    Secondary: Overall Survival

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    End point title
    		 Overall Survival
    End point description
    Results of the Kaplan-Meier analysis
    End point type
    Secondary
    End point timeframe
    Defined as time from start of abatacept treatment as trial medication (i.e. after trial registration) until death from any cause.
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20
    Units: Rates
    number (confidence interval 95%)
        Month 3
    100 (100 to 100)
        Month 6
    100 (100 to 100)
        Month 12
    94.4 (66.6 to 99.2)
        Month 15
    94.4 (66.6 to 99.2)
    No statistical analyses for this end point

    Secondary: Treatment failure

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    End point title
    		 Treatment failure
    End point description
    Defined as any premature termination of treatment for any reason.
    End point type
    Secondary
    End point timeframe
    during study
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20
    Units: Rate
        number (confidence interval 95%)
    20 (5.73 to 43.66)
    No statistical analyses for this end point

    Secondary: Cumulative steroid dose

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    End point title
    		 Cumulative steroid dose
    End point description
    14 (70.0%) of the 20 FAS patients were treated with steroids during the observation period as well as during abatacept/study drug application.
    End point type
    Secondary
    End point timeframe
    During observation period and during abatacept application
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20 [2]
    Units: Cumulative dose [mg]
    arithmetic mean (standard deviation)
        Cumulative dose during observation period
    3720 ( 2049 )
        Cumulative dose during abatacept application
    2805 ( 1312 )
    Notes
    [2] - Number of patients with at least one steroid application: 14
    No statistical analyses for this end point

    Secondary: Quality of live measured by SF36

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    End point title
    		 Quality of live measured by SF36
    End point description
    End point type
    Secondary
    End point timeframe
    Difference month 12 to screening
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20
    Units: Points
    arithmetic mean (standard deviation)
        Physical function index
    1.35 ( 18.66 )
        Role-physical index
    6.25 ( 45.18 )
        Role-emotional functioning
    -12.5 ( 50.0 )
        Social functioning index
    13.3 ( 30.4 )
        Mental health index
    2.27 ( 19.9 )
        Pain
    1.50 ( 31.6 )
        Vitality
    3.44 ( 21.5 )
        General health perception
    5.60 ( 22.75 )
        Physical component summary
    2.26 ( 11.54 )
        Mental component summary
    1.07 ( 11.65 )
    No statistical analyses for this end point

    Secondary: Clinical Global Impression-Improvement Scale (CGI-I) at month 6

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    End point title
    		 Clinical Global Impression-Improvement Scale (CGI-I) at month 6
    End point description
    The CGI-I is a 7 point scale that requires the physician to assess how much the patient’s illness has improved or worsened relative to a baseline state at the beginning of the intervention and is rated from ‘complete improvement’ to ‘worse than baseline’.
    End point type
    Secondary
    End point timeframe
    Change from the beginning of the intervention to Month 6
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20 [3]
    Units: Number of patients
        Complete improvement
    1
        Excellent improvement
    1
        Marked improvement
    0
        Moderate improvement
    6
        Minimal improvement
    4
        No change
    5
        Worse
    1
    Notes
    [3] - Number of patients valid: 18
    No statistical analyses for this end point

    Secondary: CHAI-Morbidity Score

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    End point title
    		 CHAI-Morbidity Score
    End point description
    Assessment ranged from 0 points (none) over 1 point (mild) and 2 points (moderate) to 3 points (severe).
    End point type
    Secondary
    End point timeframe
    Difference month 12 to screening
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20
    Units: CHAI score points
    arithmetic mean (standard deviation)
        Lung
    0.25 ( 1.14 )
        Gut
    -0.18 ( 1.59 )
        Cytopenia
    -0.12 ( 0.33 )
        CNS
    0 ( 0 )
        Immune system
    -2.41 ( 3.18 )
        Lymphoproliferation
    -0.88 ( 1.69 )
        Skin
    0.06 ( 1.39 )
    No statistical analyses for this end point

    Secondary: Clinical Global Impression-Improvement Scale (CGI-I) at month 12

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    End point title
    		 Clinical Global Impression-Improvement Scale (CGI-I) at month 12
    End point description
    End point type
    Secondary
    End point timeframe
    Change from the beginning of the intervention to Month 12
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20 [4]
    Units: Number of patients
        Complete improvement
    0
        Excellent improvement
    2
        Marked improvement
    3
        Moderate improvement
    4
        Minimal improvement
    1
        No change
    6
        Worse
    1
    Notes
    [4] - Number of patients valid: 17
    No statistical analyses for this end point

    Secondary: Clinical Global Impression-Improvement Scale (CGI-I) at month 15

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    End point title
    		 Clinical Global Impression-Improvement Scale (CGI-I) at month 15
    End point description
    According to CTP the visit at month 15 (follow-up visit) was performed as onsite visit only in case of termination of abatacept treatment after 12 months trial treatment (n=7)
    End point type
    Secondary
    End point timeframe
    Change from the beginning of the intervention to Month 15
    End point values
    		 Full analysis set (FAS)
    Number of subjects analysed
    20 [5]
    Units: Number of patients
        Complete improvement
    0
        Excellent improvement
    1
        Marked improvement
    0
        Moderate improvement
    0
        Minimal improvement
    0
        No change
    4
        Worse
    2
    Notes
    [5] - Number of patients valid: 7
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Complete study
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22
    Reporting groups
    Reporting group title
    Abatacept
    Reporting group description
    		 125 mg abatacept s.c. once weekly

    Serious adverse events
    		 Abatacept
    Total subjects affected by serious adverse events
         subjects affected / exposed
    9 / 20 (45.00%)
         number of deaths (all causes)
    1
         number of deaths resulting from adverse events
    1
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Acute myeloid leukaemia
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Tremor
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Lymphadenopathy
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Pancreatitis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Cholelithiasis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatic failure
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Renal and urinary disorders
    Renal tubular acidosis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Endocrine disorders
    Primary adrenal insufficiency
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Peritonitis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    COVID-19
         subjects affected / exposed
    3 / 20 (15.00%)
         occurrences causally related to treatment / all
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Metabolism and nutrition disorders
    Hyperkalaemia
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Abatacept
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    19 / 20 (95.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Anogenital warts
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Skin papilloma
         subjects affected / exposed
    2 / 20 (10.00%)
         occurrences all number
    2
    Surgical and medical procedures
    COVID-19 immunisation
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    3
    Injection site paraesthesia
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Puncture site pain
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Pyrexia
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    2
    Reproductive system and breast disorders
    Genital tract inflammation
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    2
    Dyspnoea
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Epistaxis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Oropharyngeal pain
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    2
    Throat irritation
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Tonsillar hypertrophy
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    2
    Investigations
    Blood creatinine increased
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Blood potassium decreased
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Haemoglobin decreased
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Intraocular pressure increased
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    SARS-CoV-2 test positive
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Radius fracture
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Headache
         subjects affected / exposed
    11 / 20 (55.00%)
         occurrences all number
    50
    Hyperaesthesia
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Migraine
         subjects affected / exposed
    2 / 20 (10.00%)
         occurrences all number
    2
    Blood and lymphatic system disorders
    Leukocytosis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    2
    Lymphadenopathy
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    2
    Monoclonal B-cell lymphocytosis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Thrombocytopenia
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Eye disorders
    Blepharitis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Eye inflammation
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Abdominal pain upper
         subjects affected / exposed
    2 / 20 (10.00%)
         occurrences all number
    2
    Cheilitis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Constipation
         subjects affected / exposed
    2 / 20 (10.00%)
         occurrences all number
    3
    Diarrhoea
         subjects affected / exposed
    2 / 20 (10.00%)
         occurrences all number
    3
    Abdominal pain lower
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Gastritis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    2
    Nausea
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Noninfective gingivitis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Toothache
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Hepatobiliary disorders
    Biliary colic
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Cholestasis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Alopecia
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Dermatitis psoriasiform
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Eczema
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Hidradenitis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    2
    Petechiae
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    2
    Rash
         subjects affected / exposed
    2 / 20 (10.00%)
         occurrences all number
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    5 / 20 (25.00%)
         occurrences all number
    6
    Arthritis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Back pain
         subjects affected / exposed
    2 / 20 (10.00%)
         occurrences all number
    2
    Muscle tightness
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Musculoskeletal pain
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Neck pain
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Osteoarthritis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    2
    Pain in extremity
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Infections and infestations
    Abscess
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Bronchitis
         subjects affected / exposed
    3 / 20 (15.00%)
         occurrences all number
    3
    COVID-19
         subjects affected / exposed
    2 / 20 (10.00%)
         occurrences all number
    3
    Clostridium difficile infection
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Coronavirus infection
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Folliculitis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Gastrointestinal infection
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Gastrointestinal viral infection
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Genital herpes
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    3
    Herpes zoster
         subjects affected / exposed
    2 / 20 (10.00%)
         occurrences all number
    2
    Hordeolum
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    2
    Nasopharyngitis
         subjects affected / exposed
    2 / 20 (10.00%)
         occurrences all number
    2
    Oesophageal candidiasis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Oral herpes
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    4
    Vulvovaginal candidiasis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Sinusitis
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    1
    Viral infection
         subjects affected / exposed
    1 / 20 (5.00%)
         occurrences all number
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    31 Mar 2020
    Implemented changes during initial approval process according to CA requirements: • In section 4.3, page 34 the wording of the exclusion criterion No. 21 has been adapted according to the CTFG paper, section 4.1 „Birth control methods which may be considered as highly effective“. The methods „vasectomised partner“ and „sexual abstinence“ seem not feasible to us in this clinical trial. The request to use appropriate contraceptive measures at least up to 14 weeks after the last dose of abatacept has been included in section 4.3. • In section 4.3, page 35 the definition according to the CTFG paper has been included. In section 19, page 74 the CTFG paper has been added to the list of references. In the complete Clinical Trial Protocol the wording „women in reproductive age“ has been replaced by „women of child bearing potential“. • In section 1.4, page 27 we have added further information based on unpublished data of patients with CTLA4 insufficiency treated with abatacept on local compassionate use programs.
    21 Apr 2020
    • In the synopsis, page 14 as well as in section 4.3, page 35 the exclusion criteria 8 and 9 have been changed to an overall exclusion criterion no. 8: any malignancies within the last 4 years. • In section 13.5.5, page 68 the term “treatment failure rate” was precised: defined as the number of treatment failures divided by the number of patients receiving abatacept • In section 15.3, page 70 the patients´ agreement during the informed consent procedure to “codified (“pseudonymised”) transmission to the members of the GAIN network” was deleted.
    19 Oct 2020
    - Change of LKP - elongation of screening period - Corrected: abdominal lymph nodes are measured by ultrasonography not by palpation. - Bodyplethysmography is performed for all patients during screening period to evaluate the inclusion criteria for lung involvement (in V 2.0 only for patients with lung involvement). During course of study the examination is performed only for patients with lung involvement. - Borg Dyspnoe Scala is performed for all patients (in V 2.0 only for patients with lung involvement) to check possible changes of lung function during course of study. - Completion of patient questionnaires SF36, SGRQ, IBDQ is shifted from screening to baseline visit, because this is the time point directly before application of first trial medication. - Assessment based on NANO Scale is performed for all patients during screening period to evaluate possible neurological impairment (in V 2.0 only for patients with CNS involvement). During course of study the examination is performed only for patients with CNS involvement. - Examination of skin is performed for all patients during all onsite visits to evaluate possible skin involvement (in V 2.0 only for patients with skin involvement). - Incorrect unit - Elongation of period between stopping rituximab therapy and inclusion into trial adapted according to half-life period of rituximab. - The exclusion criterion No 5 was splitted - Definition of a lag period between stopping prophylactic CMV treatment and inclusion into the trial - Decision for treatment with abatacept already was done before inclusion into the trial.
    23 Jul 2021
    - change of LKP - Adaptation to current trial status - The secondary endpoints for patients with involvement of immune system do not reflect the parameters used in the CHAI-Morbidity-Score for the Immune System. The endpoints have been adapted accordingly. - Implementation of further secondary endpoints (safety). - Inclusion criteria do not reflect the parameters used in the CHAI-Morbidity-Score for the Immune System. The inclusion criteria have been adapted accordingly. - Adaption of exclusion criteria - Addition of mandatory concomitant medication - Adaption of secondary endpoints

    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

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/36262801
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