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   44336   clinical trials with a EudraCT protocol, of which   7366   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 Phase 2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in Heterozygous Carriers of Granulin or C9orf72 Mutations Causative of Frontotemporal Dementia

    Summary
    EudraCT number
    		 2019-000138-20
    Trial protocol
    		 GB   DE   NL   IT  
    Global end of trial date
    05 Jun 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    16 May 2025
    First version publication date
    16 May 2025
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    AL001-2
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03987295
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Alector Inc.
    Sponsor organisation address
    131 Oyster Point Boulevard, Suite 600, South San Francisco, United States, CA 94080
    Public contact
    Alector Medical Information, Alector Inc., +1 650-826-2454, medinfo@alector.com
    Scientific contact
    Alector Medical Information, Alector Inc., +1 650-826-2454, medinfo@alector.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
    10 Mar 2025
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    05 Jun 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    05 Jun 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Part 1: To evaluate the safety and tolerability of intravenous (IV) administration of latozinemab over 96 weeks in asymptomatic and symptomatic carriers of a granulin (GRN) mutation causative of frontotemporal dementia (FTD) and in symptomatic carriers of a C9orf72 mutation causative of FTD. Part 2: To assess the long-term safety and tolerability of latozinemab in participants who have completed 96 weeks of treatment on Part 1 of the study.
    Protection of trial subjects
    This trial was designed and monitored in accordance with Alector procedures, which comply with the ethical principles of Good Clinical Practice (GCP) and the International Council for Harmonisation (ICH) as required by the major regulatory authorities, and in accordance with the ethical principles that have their origins in the Declaration of Helsinki.
    Background therapy
    		 The most common concomitant medications were: psycholeptics, psychoanaleptics, lipid modifying agents, and vaccines (including influenza and COVID-19).
    Evidence for comparator
    		 This was an open-label study.
    Actual start date of recruitment
    27 Sep 2019
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety
    Long term follow-up duration
    		 3 Months
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Netherlands: 4
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    Germany: 4
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    United States: 15
    Country: Number of subjects enrolled
    Canada: 5
    Worldwide total number of subjects
    33
    EEA total number of subjects
    10
    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
    10
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 A total of 45 participants enrolled of which 33 received at least one dose of AL001-2 in Part 1;12 of these participants had rolled over from Phase 1 study AL001-1. 16 participants completed Part 1 - 3 did not enroll in Part 2 and were counted as completed study, and 13 enrolled in Part 2. 9 participants completed both Part 1 and Part 2.

    Pre-assignment
    Screening details
    		 Part 1: Screening within 6 weeks prior to Day 1

    Period 1
    Period 1 title
    Part 1 + Part 2 (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    Part 1 was 96-week evaluation of safety, tolerability, PK, PD and clinical effect of latozinemab administered intravenously (60 mg/kg, every 4 weeks [q4w]) for a total of 25 doses (96-week dosing period). Part 2 was for eligible participants who had completed the 96-week Part 1 treatment period.The OLE period evaluated the long-term safety and tolerability of latozinemab administered at the same dose and regimen as Part 1 (60 mg/kg, q4w) for up to a total of 25 doses (96-week optional OLE period

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 aFTD-GRN
    Arm description
    		 aFTD-GRN - asymptomatic frontotemporal dementia with heterozygous progranulin gene mutation
    Arm type
    		 Experimental

    Investigational medicinal product name
    AL001
    Investigational medicinal product code
    Other name
    Latozinemab (human recombinant anti-human Sortilin IgG1 monoclonal antibody)
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Part 1 - Latozinemab administered intravenously (60 mg/kg, every 4 weeks [q4w]), for a total of 25 doses (96-week dosing period); Part 2 - Latozinemab administered intravenously (60 mg/kg, every 4 weeks [q4w]), for a total of 25 doses (96-week dosing period).

    Arm title
    		 FTD-GRN
    Arm description
    		 FTD-GRN - symptomatic carriers of GRN mutation causative of FTD
    Arm type
    		 Experimental

    Investigational medicinal product name
    AL001
    Investigational medicinal product code
    Other name
    Latozinemab (human recombinant anti-human Sortilin IgG1 monoclonal antibody)
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Part 1 - Latozinemab administered intravenously (60 mg/kg, every 4 weeks [q4w]), for a total of 25 doses (96-week dosing period); Part 2 - Latozinemab administered intravenously (60 mg/kg, every 4 weeks [q4w]), for a total of 25 doses (96-week dosing period).

    Arm title
    		 FTD-C9orf72
    Arm description
    		 FTD-C9orf72 - symptomatic carriers of C9orf72 hexanucleotide repeat expansion mutation causative of FTD
    Arm type
    		 Experimental

    Investigational medicinal product name
    AL001
    Investigational medicinal product code
    Other name
    Latozinemab (human recombinant anti-human Sortilin IgG1 monoclonal antibody)
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Part 1 - Latozinemab administered intravenously (60 mg/kg, every 4 weeks [q4w]), for a total of 25 doses (96-week dosing period); Part 2 - Latozinemab administered intravenously (60 mg/kg, every 4 weeks [q4w]), for a total of 25 doses (96-week dosing period).

    Number of subjects in period 1
    		aFTD-GRN FTD-GRN FTD-C9orf72
    Started
    5
    12
    16
    Completed
    5
    5
    6
    Not completed
    0
    7
    10
         Administrative or Other Reasons
    -
    2
    2
         Withdrawal by Participant
    -
    4
    6
         Death
    -
    -
    1
         Investigator Discretion
    -
    1
    -
         Long-Term care facility placement and requires con
    -
    -
    1

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    aFTD-GRN
    Reporting group description
    		 aFTD-GRN - asymptomatic frontotemporal dementia with heterozygous progranulin gene mutation

    Reporting group title
    FTD-GRN
    Reporting group description
    		 FTD-GRN - symptomatic carriers of GRN mutation causative of FTD

    Reporting group title
    FTD-C9orf72
    Reporting group description
    		 FTD-C9orf72 - symptomatic carriers of C9orf72 hexanucleotide repeat expansion mutation causative of FTD

    Reporting group values
    		 aFTD-GRN FTD-GRN FTD-C9orf72 Total
    Number of subjects
    		 5 12 16 33
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 5 7 11 23
        From 65-84 years
    		 0 5 5 10
    Age continuous
    Units: years
        median (full range (min-max))
    		 59.0 (32 to 63) 59.5 (48 to 78) 60.0 (40 to 74) -
    Gender categorical
    Units: Subjects
        Female
    		 1 4 9 14
        Male
    		 4 8 7 19

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    aFTD-GRN
    Reporting group description
    		 aFTD-GRN - asymptomatic frontotemporal dementia with heterozygous progranulin gene mutation

    Reporting group title
    FTD-GRN
    Reporting group description
    		 FTD-GRN - symptomatic carriers of GRN mutation causative of FTD

    Reporting group title
    FTD-C9orf72
    Reporting group description
    		 FTD-C9orf72 - symptomatic carriers of C9orf72 hexanucleotide repeat expansion mutation causative of FTD

    Primary: Duration of exposure to study drug (days)

    		 Close Top of page
    End point title
    		 Duration of exposure to study drug (days) [1]
    End point description
    The median exposure was 700 days (23 months) for the whole Safety Population. The median exposure was 1406 days (46 months) in the aFTD-GRN cohort, 498 days (16 months) in the FTD-GRN cohort, and 675 (22 months) days in the FTD-C9orf72 cohort.
    End point type
    Primary
    End point timeframe
    Part 1 - 96 weeks + Part 2 - 96 weeks
    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: There is no statistical analysis for this primary end point.
    End point values
    		 aFTD-GRN FTD-GRN FTD-C9orf72
    Number of subjects analysed
    5
    12
    16
    Units: day
    median (full range (min-max))
        Duration of exposure to study drug (days)
    1406.0 (1398 to 1407)
    498.0 (111 to 1395)
    675.0 (28 to 1407)
    No statistical analyses for this end point

    Primary: Severity of TEAEs

    		 Close Top of page
    End point title
    		 Severity of TEAEs [2]
    End point description
    In total, 31 out of 33 (93.9%) participants experienced a TEAE of which 75.8% had mild or moderate TEAEs in severity; no treatment-related TEAEs were considered severe (or worse) in severity. There were no treatment-related TESAEs observed in the study and of the 6 TESAEs, one was fatal.
    End point type
    Primary
    End point timeframe
    Part 1 - 96 weeks + Part 2 - 96 weeks
    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: There is no statistical analysis for this primary end point.
    End point values
    		 aFTD-GRN FTD-GRN FTD-C9orf72
    Number of subjects analysed
    5
    12
    16
    Units: Event
    number (not applicable)
        Mild
    43
    58
    84
        Moderate
    4
    9
    24
        Severe
    3
    1
    4
        Life Threatening
    0
    0
    0
        Death
    0
    0
    1
    No statistical analyses for this end point

    Primary: Severity of Treatment-Related TEAEs

    		 Close Top of page
    End point title
    		 Severity of Treatment-Related TEAEs [3]
    End point description
    End point type
    Primary
    End point timeframe
    Part 1 - 96 weeks + Part 2 - 96 weeks
    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: There is no statistical analysis for this primary end point.
    End point values
    		 aFTD-GRN FTD-GRN FTD-C9orf72
    Number of subjects analysed
    5
    12
    16
    Units: Event
    number (not applicable)
        Mild
    5
    1
    12
        Moderate
    1
    1
    5
        Severe
    0
    0
    0
        Life Threatening
    0
    0
    0
        Death
    0
    0
    0
    No statistical analyses for this end point

    Primary: Any TEAE Leading to Study Drug Discontinuation

    		 Close Top of page
    End point title
    		 Any TEAE Leading to Study Drug Discontinuation [4]
    End point description
    Two TEAEs led to latozinemab discontinuation. One participant in the FTD-GRN cohort with a medical history of mitral valve prolapse and incompetence had worsening that led to a valvuloplasty procedure and withdrawal from the study. One participant in the FTD-C9orf72 cohort experienced progression of ALS and was admitted to hospice, discontinuing the study.
    End point type
    Primary
    End point timeframe
    Part 1 - 96 weeks + Part 2 - 96 weeks
    Notes
    [4] - 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: There is no statistical analysis for this primary end point.
    End point values
    		 aFTD-GRN FTD-GRN FTD-C9orf72
    Number of subjects analysed
    5
    12
    16
    Units: Event
    number (not applicable)
        Any TEAE Leading to Study Drug Discontinuation
    0
    1
    1
    No statistical analyses for this end point

    Primary: Immunogenicity Antidrug Antibodies (ADA)

    		 Close Top of page
    End point title
    		 Immunogenicity Antidrug Antibodies (ADA) [5]
    End point description
    Across all cohorts, the ADA positivity rate ranged from 3% (3/33) to 33% (11/33) across dosing visits. In aFTD-GRN cohort, 3 out of 5 (60%) participants had ADA 2 weeks after the first dose. In FTD-GRN cohort, 3 out of 12 (25.0%) participants had ADA 2 weeks after the first dose; in FTD-C9orf72 cohort, 3 out of 16 (18.8%) participants had ADA 2 weeks after the first dose. Only 1 out of 33 (3%) participants was ADA positive before the 2nd dose administration. After multiple dose administration, the highest ADA positivity rate, 24.2% (8/33), was found at Week 13 after every 4 weeks of administration. After 52 weeks of treatment with latozinemab, only 2 out of 33 (6.1%) participants were ADA-positive across all cohorts.Median (min-max) ADA titer was 40 (20-5120) at 2 weeks after the first dose and was 240 (160-320) at Week 52.The higher incidence of ADA 2 weeks after the first dose could be probably due to the fact that 12 of these participants had rolled over from Phase 1 study AL001-1.
    End point type
    Primary
    End point timeframe
    Part 1 - 96 weeks + Part 2 - 96 weeks
    Notes
    [5] - 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: There is no statistical analysis for this primary end point.
    End point values
    		 aFTD-GRN FTD-GRN FTD-C9orf72
    Number of subjects analysed
    5
    12
    16
    Units: percent
    median (full range (min-max))
        Week 2 - ADA Titer
    320.0 (20 to 5120)
    20.0 (20 to 2560)
    40.0 (20 to 160)
        Week 13 - ADA Titer
    160.0 (20 to 2560)
    160.0 (160 to 640)
    120.0 (80 to 160)
        Week 53 - ADA Titer
    0 (0 to 0)
    320.0 (320 to 320)
    160.0 (160 to 160)
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Latozinemab has an acceptable safety profile and was well-tolerated in participants carrying GRN or C9orf72 mutation causative of FTD.
    Adverse event reporting additional description
    In total, 31 out of 33 (93.9%) participants experienced a TEAE of which 75.8% had mild or moderate TEAEs in severity; no treatment-related TEAEs were considered severe (or worse) in severity. There were no treatment-related TESAEs observed in the study and of the 6 TESAEs, one was fatal.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.1.
    Reporting groups
    Reporting group title
    aFTD-GRN
    Reporting group description
    		 -

    Reporting group title
    FTD-GRN
    Reporting group description
    		 -

    Reporting group title
    FTD-C9orf72
    Reporting group description
    		 -

    Serious adverse events
    		 aFTD-GRN FTD-GRN FTD-C9orf72
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 5 (20.00%)
    3 / 12 (25.00%)
    2 / 16 (12.50%)
         number of deaths (all causes)
    0
    0
    1
         number of deaths resulting from adverse events
    0
    0
    1
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Lip and/or oral cavity cancer
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Procedural pneumothorax
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Deep vein thrombosis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Amyotrophic lateral sclerosis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    2 / 16 (12.50%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    Syncope
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Aggression
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 0
    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
    		 aFTD-GRN FTD-GRN FTD-C9orf72
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 5 (80.00%)
    9 / 12 (75.00%)
    13 / 16 (81.25%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Haemangioma of skin
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Seborrhoeic keratosis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Vascular disorders
    Diastolic hypertension
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Hypertension
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Subclavian artery occlusion
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    General disorders and administration site conditions
    Administration site extravasation
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Crying
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Fatigue
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Influenza like illness
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Infusion site extravasation
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Infusion site pain
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Infusion site paraesthesia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Epistaxis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Nasal congestion
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Actinic keratosis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 12 (8.33%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    1
    Depression
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Hallucination, auditory
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Illusion
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Impulsive behaviour
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Insomnia
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 12 (16.67%)
    0 / 16 (0.00%)
         occurrences all number
    0
    2
    0
    Obsessive-compulsive disorder
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Paranoia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Restlessness
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Sleep disorder
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Product issues
    Device physical property issue
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Investigations
    Blood cholesterol increased
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Blood creatine phosphokinase increased
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Blood potassium increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Hepatic enzyme increased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Lipase increased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Weight decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Injury, poisoning and procedural complications
    Alcohol poisoning
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Arthropod bite
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Contusion
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    1
    0
    1
    Fall
         subjects affected / exposed
    2 / 5 (40.00%)
    5 / 12 (41.67%)
    7 / 16 (43.75%)
         occurrences all number
    2
    5
    7
    Head injury
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Joint injury
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Patella fracture
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Post lumbar puncture syndrome
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 12 (16.67%)
    0 / 16 (0.00%)
         occurrences all number
    0
    2
    0
    Procedural pain
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Procedural pneumothorax
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Road traffic accident
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Skin abrasion
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Skin laceration
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Sunburn
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Thermal burn
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Weight increased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Aortic valve stenosis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Mitral valve incompetence
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Nervous system disorders
    Aphasia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Carotid artery stenosis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Dizziness
         subjects affected / exposed
    2 / 5 (40.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    2
    0
    0
    Headache
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 12 (8.33%)
    2 / 16 (12.50%)
         occurrences all number
    1
    1
    2
    Hypoaesthesia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Lacunar infarction
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Lacunar stroke
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Myoclonus
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Paraesthesia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Parkinsonian rest tremor
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Peripheral sensory neuropathy
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Polyneuropathy
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Seizure like phenomena
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Subdural hygroma
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Syncope
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 12 (16.67%)
    1 / 16 (6.25%)
         occurrences all number
    0
    2
    1
    Blood and lymphatic system disorders
    Leukopenia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Thrombocytopenia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Eye disorders
    Blepharitis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Dry eye
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Episcleritis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Lacrimation increased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Retinal detachment
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Vision blurred
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Abdominal pain
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Constipation
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 12 (16.67%)
    1 / 16 (6.25%)
         occurrences all number
    0
    2
    1
    Diarrhoea
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Dry mouth
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Dysphagia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Inguinal hernia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Nausea
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    1
    0
    1
    Pancreatitis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Toothache
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Vomiting
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Hepatobiliary disorders
    Cholelithiasis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Dermatitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Dermatitis contact
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    2 / 16 (12.50%)
         occurrences all number
    0
    1
    2
    Ecchymosis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Intertrigo
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Photodermatosis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Pseudofolliculitis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Psoriasis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Rash
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 12 (8.33%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    1
    Seborrhoeic dermatitis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Renal and urinary disorders
    Micturition urgency
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Nocturia
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Pollakiuria
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 12 (16.67%)
    0 / 16 (0.00%)
         occurrences all number
    0
    2
    0
    Urge incontinence
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Urinary retention
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Endocrine disorders
    Goitre
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Hypothyroidism
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    2 / 5 (40.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    2
    0
    0
    Bursitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Costochondritis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Medial tibial stress syndrome
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Muscular weakness
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Musculoskeletal stiffness
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Myalgia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    1
    0
    1
    Osteoarthritis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Tendon disorder
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Trigger finger
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Infections and infestations
    Acute sinusitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Asymptomatic COVID-19
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Bronchitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    COVID-19
         subjects affected / exposed
    2 / 5 (40.00%)
    0 / 12 (0.00%)
    5 / 16 (31.25%)
         occurrences all number
    2
    0
    5
    Cellulitis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Ear infection
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Eye infection bacterial
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Folliculitis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Herpes simplex
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Hordeolum
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Lyme disease
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    1
    1
    0
    Onychomycosis
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Otitis externa
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Pneumonia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    Skin infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Tooth infection
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 5 (40.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    2
    0
    1
    Urinary tract infection
         subjects affected / exposed
    1 / 5 (20.00%)
    2 / 12 (16.67%)
    6 / 16 (37.50%)
         occurrences all number
    1
    2
    6
    Viral rhinitis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Vulvovaginal mycotic infection
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 12 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    Eye contusion
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 12 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    Metabolism and nutrition disorders
    Abnormal loss of weight
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Gout
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 12 (8.33%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    09 Apr 2019
    Protocol Version 1.0 - Original Protocol
    22 Apr 2019
    Protocol Version 1.1 Removed UPDRS Part III Clinical Outcome Assessment (COA), replaced “PPD” with “the CRO”, and corrected inclusion/exclusion criteria numbering
    24 Jun 2019
    Protocol Version 1.2 Additional criteria added for withdrawal from study drug; language around infusion and injection-related reactions updated to align with Investigator’s Brochure; clarification on AE and SAE reporting
    02 Aug 2019
    Protocol Version 1.3 Inclusion of independent Data Monitoring Committee (DMC)
    12 Sep 2019
    Protocol Version 1.4 Expanded risk/benefit assessment; clarification to the language regarding withdrawal from study and withdrawal from study drug
    26 Sep 2019
    Protocol Version 2.0 Updated terminology of Clinical Outcomes Assessments (COAs); addition of optional Open Label Extension (OLE), clarification of inclusion/exclusion criteria, updates to Schedule of Assessments
    13 Dec 2019
    Protocol Version 1.5 Administrative updates to the study team and clarification on reporting timeline for SAEs
    02 Jun 2020
    Protocol Version 3.0 Duration of treatment period expanded from 46 to 96 weeks; update to number of planned participants; updates to Schedule of Assessments and Statistical Analyses; addition of appendix to describe risk review and adaptations as a result of COVID-19
    13 Jul 2020
    Protocol Version 3.1 Update to AE definitions and pregnancy reporting instructions; update to COVID-19 pandemic language; minor formatting changes
    23 Nov 2020
    Protocol Version 3.2 Background information updated based on current IB; addition of protocol-specific instructions for reporting AEs related to disease progression
    04 Feb 2021
    Protocol Version 4.0 Added a Part 2 optional OLE period including updated study objectives, endpoints and assessments; updated relevant section with data from completed trial; safety follow-up period changed from 8-weeks to 10-weeks

    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 May 17 02:39:33 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA