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    Clinical Trial Results:
    Phase IIa Biomarker Study to Evaluate the Efficacy, Safety and Tolerability of AT-1 in Patients with Hereditary Cystatin C Amyloid Angiopathy (HCCAA) - the AT1-HCCAA study

    Summary
    EudraCT number
    		 2017-004776-56
    Trial protocol
    		 IS  
    Global end of trial date
    03 Sep 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    13 Oct 2021
    First version publication date
    13 Oct 2021
    Other versions
    Summary report(s)
    		 AT NAC Phase II Synopsis

    Trial information

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    Trial identification
    Sponsor protocol code
    AT1-2017
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Arctic Therapeutics ehf.
    Sponsor organisation address
    Sóltún 11, Reykjavík, Iceland, 105
    Public contact
    Head, Clinical Development, Arctic Therapeutics ehf., + 1 2674554534 , hakon@hakonarson.com
    Scientific contact
    Head, Clinical Development, Arctic Therapeutics ehf., + 1 2674554534 , hakon@hakonarson.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
    01 Sep 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    03 Sep 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    03 Sep 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    • Evaluate safety and tolerability of AT-1 administered orally in adults (ages 18 and over) with HCCAA with or without dementia symptoms • Assess dose-response relationship of AT-1 on HCCAA disease progression, including • Biomarker response from skin biopsies (reduction in cystatin C stain) • Assessment of cognitive status using dementia rating scales
    Protection of trial subjects
    Independent data monitoring committee (IDMC) was established to review and oversee any issues with trial subjects. Clinical trial protocol was designed to minimize pain and distress of trial subjects.
    Background therapy
    		 None.
    Evidence for comparator
    		 N/A. No comparators used in this study.
    Actual start date of recruitment
    29 Jun 2018
    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
    Iceland: 17
    Worldwide total number of subjects
    17
    EEA total number of subjects
    17
    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)
    14
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Subjects were all recruited from Iceland, starting on July 1, 2019 through November 28, 2019. Adults (18+ years old) who were suspected carriers of the L68Q mutation in Cystatin C were invited to participate.

    Pre-assignment
    Screening details
    		 All adult subjects (18+ years old) were invited to participate in the study. Overall, 22 subjects were screened, and 5 were excluded when confirmation genetic testing revealed they were not carriers or L68Q mutation.

    Pre-assignment period milestones
    Number of subjects started
    		 22 [1]
    Number of subjects completed
    		 17

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 Did not carry L68Q mutation: 5
    Notes
    [1] - The number of subjects reported to have started the pre-assignment period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: 22 subjects screened, of whom 17 were officially enrolled. 5 were excluded since they failed screening due to absence of L68Q mutation.
    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    N/A. Open label study.

    Arms
    Arm title
    		 All subjects
    Arm description
    		 All enrolled subjects dosed daily with active drug in this open label study.
    Arm type
    		 Experimental

    Investigational medicinal product name
    N-acetyl cystein
    Investigational medicinal product code
    AT-1
    Other name
    Mucolysin, acetylcystein
    Pharmaceutical forms
    Effervescent tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1200mg of AT-1 administered bid (twice daily) per os (orally) via soluble effervescent tablets (600mg each tablet).

    Number of subjects in period 1
    		All subjects
    Started
    17
    Completed
    15
    Not completed
    2
         Pregnancy
    2

    Baseline characteristics

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

    Reporting group values
    		 Overall trial Total
    Number of subjects
    		 17 17
    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)
    		 14 14
        From 65-84 years
    		 3 3
        85 years and over
    		 0 0
    Gender categorical
    Units: Subjects
        Female
    		 11 11
        Male
    		 6 6

    End points

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    End points reporting groups
    Reporting group title
    All subjects
    Reporting group description
    		 All enrolled subjects dosed daily with active drug in this open label study.

    Primary: Dementia rating scale

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    End point title
    		 Dementia rating scale [1]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline and 3, 6, and 9 months after treatment with AT-001.
    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: No notable decline/change in cognitive function throughout study based on physicians' assessments.
    End point values
    		 All subjects
    Number of subjects analysed
    17
    Units: Rating scale score
    arithmetic mean (standard deviation)
        Total score, baseline
    135.6 ± 10.72
        Total score, 3 months
    133.93 ± 11.27
        Total score, 6 months
    137.53 ± 12.23
        Total score, 9 months
    133.33 ± 12.51
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline and 3, 6, and 9 months after treatment with AT-001
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    All treated subjects
    Reporting group description
    		 -

    Serious adverse events
    		 All treated subjects
    Total subjects affected by serious adverse events
         subjects affected / exposed
    8 / 17 (47.06%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Vascular disorders
    Hemorrage
    Additional description: Small intracranial hemorrhage observed upon CT scan, consistent with natural history of HCCAA disease.
         subjects affected / exposed
    5 / 17 (29.41%)
         occurrences causally related to treatment / all
    0 / 6
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Migraine
    Additional description: Headache/migraine that had worsened after a fall, but then resolved on its own.
         subjects affected / exposed
    1 / 17 (5.88%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Reproductive system and breast disorders
    Pregnancy
    Additional description: Subject became pregnant during course of study, initially detected by urine pregnancy test then confirmed. Withdrew from study.
         subjects affected / exposed
    2 / 17 (11.76%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 All treated subjects
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 17 (88.24%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 17 (17.65%)
         occurrences all number
    3
    General disorders and administration site conditions
    Dry mouth
         subjects affected / exposed
    2 / 17 (11.76%)
         occurrences all number
    2
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 17 (11.76%)
         occurrences all number
    2
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    5 / 17 (29.41%)
         occurrences all number
    5
    Sleep disturbance
         subjects affected / exposed
    2 / 17 (11.76%)
         occurrences all number
    2
    Infections and infestations
    Influenza
         subjects affected / exposed
    2 / 17 (11.76%)
         occurrences all number
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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
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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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