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    Clinical Trial Results:
    A randomized, double-blind, placebo-controlled, parallel-group proof of concept study to evaluate the effect of AFQ056 in obsessive compulsive disorder (OCD) patients resistant to Selective Serotonin Reuptake Inhibitor (SSRI) therapy

    Summary
    EudraCT number
    		 2012-005000-17
    Trial protocol
    		 DE   GB   CZ   BG  
    Global end of trial date
    11 Nov 2014

    Results information
    Results version number
    		 v1(current)
    This version publication date
    30 Jun 2016
    First version publication date
    30 Jun 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CAFQ056A2225
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01813019
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111,
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111,
    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
    11 Nov 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    11 Nov 2014
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To assess the effects of AFQ056 (16 weeks treatment), using Yale - Brown Obsessive Compulsive Scale (Y-BOCS) change from baseline compared to placebo
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    19 Nov 2013
    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
    Bulgaria: 19
    Country: Number of subjects enrolled
    Czech Republic: 2
    Country: Number of subjects enrolled
    Germany: 8
    Country: Number of subjects enrolled
    Poland: 4
    Country: Number of subjects enrolled
    Switzerland: 1
    Country: Number of subjects enrolled
    United States: 16
    Worldwide total number of subjects
    50
    EEA total number of subjects
    33
    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)
    49
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study consisted of a screening period of up to 35 days, a Baseline evaluation, a treatment period of 19 weeks and an end of study evaluation approximately 14 days after the last study drug administration. The total duration for each patient in the study, was approximately 27 weeks.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Matching Placebo b.i.d. dosing
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Following baseline, approximately 60 patients who are considered eligible will be randomized to Placebo arm and will receive the dosing regimen of 4 weeks Placebo b.i.d up-titration period of 50 mg,100 mg, 150 mg and 200 mg , followed by 12 weeks Placebo 200 mg fixed dose* and then a 3 week down-titration of 100 mg, 50 mg and 25 mg Placebo b.i.d) *patients that do not tolerate 200 matching placebo AFQ056 mg b.i.d may be down-titrated to 150 mg b.i.d.

    Arm title
    		 AFQ056
    Arm description
    		 AFQ056 b.i.d up-titration of 50mg,100mg, 150mg and 200 mg for 4 weeks then AFQ056 200mg b.i.d for 12 weeks and then a down-titration of AFQ056 100mg, 50mg and 25mg b.i.d for 3 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    AFQ056
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Following baseline, approximately 60 patients who are considered eligible will be randomized to AFQ056 arm and will receive the dosing regimen of 4 weeks AFQ056 b.i.d up-titration period of 50 mg,100 mg, 150 mg and 200 mg , followed by

    Number of subjects in period 1
    		Placebo AFQ056
    Started
    24
    26
    Completed
    17
    20
    Not completed
    7
    6
         Consent withdrawn by subject
    3
    2
         Protocol Deviation
    2
    1
         Adverse event, non-fatal
    -
    3
         administrative problems
    2
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Matching Placebo b.i.d. dosing

    Reporting group title
    AFQ056
    Reporting group description
    		 AFQ056 b.i.d up-titration of 50mg,100mg, 150mg and 200 mg for 4 weeks then AFQ056 200mg b.i.d for 12 weeks and then a down-titration of AFQ056 100mg, 50mg and 25mg b.i.d for 3 weeks

    Reporting group values
    		 Placebo AFQ056 Total
    Number of subjects
    		 24 26 50
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 24 25 49
        From 65-84 years
    		 0 1 1
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 39.9 ( 10.06 ) 41.3 ( 13.27 ) -
    Gender, Male/Female
    Units: participant
        Male
    		 11 13 24
        Female
    		 13 13 26

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Matching Placebo b.i.d. dosing

    Reporting group title
    AFQ056
    Reporting group description
    		 AFQ056 b.i.d up-titration of 50mg,100mg, 150mg and 200 mg for 4 weeks then AFQ056 200mg b.i.d for 12 weeks and then a down-titration of AFQ056 100mg, 50mg and 25mg b.i.d for 3 weeks

    Primary: Yale - Brown Obsessive Compulsive Scale (Y-BOCS) absolute change from baseline at Week 17 (end of 16-week dosing).

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    End point title
    		 Yale - Brown Obsessive Compulsive Scale (Y-BOCS) absolute change from baseline at Week 17 (end of 16-week dosing).
    End point description
    The Y-BOCS is a 10 item clinician-rated scale used to both determine the severity of OCD and to monitor symptom improvement throughout the course of the study. The Y-BOCS, specifically measures the severity of symptoms of OCD without being biased towards the type of obsessions or compulsions present. The scale includes questions about the amount of time spent on, how much impairment or distress experienced from, and how much resistance and control over these obsessive thoughts and compulsions. Each item is rated from 0 ("no symptoms") to 4 ("extreme symptoms") and yields a total possible score range from 0 to 40, with the following ranges indicating degree of severity: 0–7 = sub-clinical 8–15 = mild 16–23 = moderate 24–31 = severe 32–40 = extreme Baseline was compared to week 17 (end of week 16 dosing) to produce an absolute change.
    End point type
    Primary
    End point timeframe
    baseline, week 17
    End point values
    		 Placebo AFQ056
    Number of subjects analysed
    17
    21
    Units: Scores on a scale
        least squares mean (standard error)
    -8 ( 1.78 )
    -6.9 ( 1.75 )
    Statistical analysis title
    		 Yale-Brown Obsessive Compulsive Scale Change
    Comparison groups
    AFQ056 v Placebo
    Number of subjects included in analysis
    38
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.671
    Method
    		 Mixed models analysis
    Confidence interval

    Secondary: Y-BOCS reduction in total score from Baseline

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    End point title
    		 Y-BOCS reduction in total score from Baseline
    End point description
    If a subject demonstrates at least 25% reduction in total Y-BOCS from Baseline then they will be classed as a responder whereas if a subject has a reduction in Y-BOCS of less than 25% then they will be categorized as a nonresponder.
    End point type
    Secondary
    End point timeframe
    16 weeks
    End point values
    		 Placebo AFQ056
    Number of subjects analysed
    0 [1]
    0 [2]
    Units: total score
        number (not applicable)
    Notes
    [1] - Study terminated due to interim analysis. Secondary analysis not conducted.
    [2] - Study terminated due to interim analysis. Secondary analysis not conducted.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit.
    Adverse event reporting additional description
    AE additional description
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.0
    Reporting groups
    Reporting group title
    AFQ056
    Reporting group description
    		 AFQ056 b.i.d up-titration of 50mg,100mg, 150mg and 200 mg for 4 weeks then AFQ056 200mg b.i.d for 12 weeks and then a down-titration of AFQ056 100mg, 50mg and 25mg b.i.d for 3 weeks

    Reporting group title
    Placebo
    Reporting group description
    		 Matching Placebo b.i.d. dosing

    Serious adverse events
    		 AFQ056 Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 26 (3.85%)
    2 / 24 (8.33%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Injury, poisoning and procedural complications
    Face injury
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Head injury
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Limb injury
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    VIIth nerve paralysis
         subjects affected / exposed
    1 / 26 (3.85%)
    0 / 24 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Otitis media
         subjects affected / exposed
    1 / 26 (3.85%)
    0 / 24 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 AFQ056 Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    18 / 26 (69.23%)
    15 / 24 (62.50%)
    Investigations
    Aspartate aminotransferase increased
         subjects affected / exposed
    2 / 26 (7.69%)
    1 / 24 (4.17%)
         occurrences all number
    2
    1
    Cardiac disorders
    Bradycardia
         subjects affected / exposed
    0 / 26 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    2
    Nervous system disorders
    Disturbance in attention
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 24 (0.00%)
         occurrences all number
    2
    0
    Dizziness
         subjects affected / exposed
    5 / 26 (19.23%)
    2 / 24 (8.33%)
         occurrences all number
    7
    4
    Headache
         subjects affected / exposed
    10 / 26 (38.46%)
    8 / 24 (33.33%)
         occurrences all number
    18
    13
    Migraine
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 24 (0.00%)
         occurrences all number
    3
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    2 / 26 (7.69%)
    1 / 24 (4.17%)
         occurrences all number
    2
    1
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    2 / 26 (7.69%)
    1 / 24 (4.17%)
         occurrences all number
    3
    1
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    2 / 26 (7.69%)
    1 / 24 (4.17%)
         occurrences all number
    2
    2
    Dyspepsia
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 24 (0.00%)
         occurrences all number
    2
    0
    Psychiatric disorders
    Abnormal dreams
         subjects affected / exposed
    2 / 26 (7.69%)
    1 / 24 (4.17%)
         occurrences all number
    2
    1
    Agitation
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 24 (0.00%)
         occurrences all number
    5
    0
    Depression
         subjects affected / exposed
    1 / 26 (3.85%)
    2 / 24 (8.33%)
         occurrences all number
    1
    3
    Insomnia
         subjects affected / exposed
    6 / 26 (23.08%)
    2 / 24 (8.33%)
         occurrences all number
    8
    3
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 24 (0.00%)
         occurrences all number
    2
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    2 / 26 (7.69%)
    4 / 24 (16.67%)
         occurrences all number
    2
    6

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    07 May 2013
    This amendment was issued prior to patient enrollment in the trial to respond to Investigators feedback to the protocol whereby the following point was addressed: Exclusion criterion 4 “History of failure to one augmentation therapy (e.g. an increase in dose of SSRI)” was removed from the protocol. In addition, as a result of Investigator feedback, inclusion criteria 9 related to the exclusion of patients with past medical history of bipolar disorder, schizophrenia or other psychotic disorders, schizoaffective disorder, autism, borderline personality disorder, or Gilles de laTourette syndrome was amended to allow patients with high functioning ASD, e.g. Asperger’s Syndrome to be included in the study. Obsessive-compulsive disorder (OCD) and social anxiety disorder frequently co-occur in persons with ASD (Cath et al 2008). Therefore, it would be appropriate to include these patients in the study.
    07 Feb 2014
    This amendment was issued when there were two patients on the treatment and 13 were screened. Issued to respond to Investigators feedback from active study sites in light of the OCD population they were screening. •Inclusion criterion was modified to allow patients with a Y-BOCS score ≥16 to enter the study. The inclusion criterion was removed from entry criteria of the protocol. •Exclusion criterion 5 was removed from entry criteria of the protocol. •Exclusion criterion 8 was amended to allow Gilles de la Tourette syndrome patients to be included in the study, provided the primary diagnosis is OCD. Exclusion criterion 9 “History of substance dependence and/or substance abuse in the last 6 months prior to Screening, with the exception of nicotine has been amended to exclude nicotine “with the exception of nicotine” in order to be in line with Exclusion criteria 9 which was exclusion of the smokers. •Exclusion criterion 28 was amended in accordance with the AFQ056 Investigator Brochure (version 11 dated Jun-18-2013), confirming that contraception requirement for women of child-bearing potential changed from highly effective to effective.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    09 Jun 2014
    In June 2014, the Company has decided to proceed with an interim analysis based on the data from all completed patients at week 16. The company has therefore placed recruitment on hold from June 9th onwards until the results from this interim analysis become available.
    -

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    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.

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