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    Clinical Trial Results:
    A Phase 2a, Open-label, Single-arm, 2-Part Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis.

    Summary
    EudraCT number
    		 2020-000185-42
    Trial protocol
    		 GB   FR  
    Global end of trial date
    09 Dec 2021

    Results information
    Results version number
    		 v2(current)
    This version publication date
    27 Jul 2023
    First version publication date
    25 Dec 2022
    Other versions
    		 v1
    Version creation reason
    • New data added to full data set
    Update to match CT.gov

    Trial information

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    Trial identification
    Sponsor protocol code
    VX19-147-101
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04340362
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Vertex Pharmaceuticals Incorporated
    Sponsor organisation address
    50 Northern Avenue, Boston, Massachusetts, United States,
    Public contact
    Medical Monitor, Vertex Pharmaceuticals Incorporated, +1 617-341-6777, medicalinfo@vrtx.com
    Scientific contact
    Medical Monitor, Vertex Pharmaceuticals Incorporated, +1 617-341-6777, medicalinfo@vrtx.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
    14 Feb 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    11 Nov 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    09 Dec 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy, safety and pharmacokinetics (PK) of VX-147 in subjects with apolipoprotein L1 (APOL1)-mediated focal segmental glomerulosclerosis (FSGS).
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles stated in the Declaration of Helsinki and the International Conference on Harmonization (ICH) Guideline for Good Clinical Practice (GCP).
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    08 Jun 2020
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    United States: 11
    Country: Number of subjects enrolled
    United Kingdom: 3
    Worldwide total number of subjects
    16
    EEA total number of subjects
    2
    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)
    15
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This study was planned in 2 parts: Part A (Treatment Period), which consisted of 2 cohorts (i.e., cohort 1 and 2) and Part B (Optional Exploratory Off-treatment Follow-up Period). The primary and secondary efficacy analyses and safety analyses were planned for only Part A.

    Pre-assignment
    Screening details
    		 This study was conducted on adult subjects who had APOL1-mediated focal segmental glomerulosclerosis (FSGS).

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

    Arms
    Arm title
    		 VX-147
    Arm description
    		 All subjects received VX-147 at a dosage of 15 mg once daily (qd) for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included subjects with urine protein to creatinine ratio (UPCR) approximately greater than or equal to (≥) 3 g/g (± 10%) and less than (<) 10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included subjects with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Inaxaplin
    Investigational medicinal product code
    VX-147
    Other name
    IXP
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subject received VX-147 once daily.

    Number of subjects in period 1
    		VX-147
    Started
    16
    Cohort 1
    3 [1]
    Cohort 2
    13 [2]
    Completed
    15
    Not completed
    1
         Withdrawal of consent (not due to adverse event)
    1
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The milestone represents the number of subjects in Cohort 1.
    [2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The milestone represents the number of subjects in Cohort 2.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    VX-147
    Reporting group description
    		 All subjects received VX-147 at a dosage of 15 mg once daily (qd) for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included subjects with urine protein to creatinine ratio (UPCR) approximately greater than or equal to (≥) 3 g/g (± 10%) and less than (<) 10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included subjects with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2.

    Reporting group values
    		 VX-147 Total
    Number of subjects
    		 16 16
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 38.8 ( 14.5 ) -
    Gender categorical
    Units: Subjects
        Female
    		 9 9
        Male
    		 7 7
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 0
        Not Hispanic or Latino
    		 16 16
        Unknown or Not Reported
    		 0 0
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0
        Asian
    		 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0
        Black or African American
    		 16 16
        White
    		 0 0
        More than one Race
    		 0 0
        Unknown or Not Reported
    		 0 0
    Urine Protein to Creatinine Ratio (UPCR)
    Units: g/g
        arithmetic mean (standard deviation)
    		 2.08 ( 0.90 ) -

    End points

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    End points reporting groups
    Reporting group title
    VX-147
    Reporting group description
    		 All subjects received VX-147 at a dosage of 15 mg once daily (qd) for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included subjects with urine protein to creatinine ratio (UPCR) approximately greater than or equal to (≥) 3 g/g (± 10%) and less than (<) 10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included subjects with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2.

    Primary: Percent Change From Baseline in UPCR

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    End point title
    		 Percent Change From Baseline in UPCR [1]
    End point description
    End point type
    Primary
    End point timeframe
    From Baseline up to Week 13
    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: This is a single arm study. Only descriptive statistics for geometric mean percent change from baseline were planned . No statistical comparisons were planned for this endpoint.
    End point values
    		 VX-147
    Number of subjects analysed
    13
    Units: Percent Change
    geometric mean (confidence interval 95%)
        Cohort 1 (n=3)
    -47.7 (-70.1 to -8.5)
        Cohort 2 (n=10)
    -47.5 (-63.4 to -24.6)
        Total (n=13)
    -47.6 (-60.0 to -31.3)
    No statistical analyses for this end point

    Secondary: Safety and Tolerability as Assessed by Number of Subjects With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

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    End point title
    		 Safety and Tolerability as Assessed by Number of Subjects With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
    End point description
    Safety set included all subjects who received at least 1 dose of study drug in the treatment period. Here, the "n" signifies subjects who were evaluable for the specified cohorts.
    End point type
    Secondary
    End point timeframe
    From Baseline up to Week 17
    End point values
    		 VX-147
    Number of subjects analysed
    16
    Units: Subjects
        Cohort 1 : Subjects with TEAEs (n=3)
    3
        Cohort 1 : Subjects with SAEs (n=3)
    0
        Cohort 2 : Subjects with TEAEs (n=13)
    12
        Cohort 2 : Subjects with SAEs (n=13)
    1
    No statistical analyses for this end point

    Secondary: Maximum Observed Concentration (Cmax) of VX-147

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    End point title
    		 Maximum Observed Concentration (Cmax) of VX-147
    End point description
    Pharmacokinetic (PK) set included all subjects who have received at least 1 dose of study drug in the treatment period. Here, the "n" signifies subjects who were evaluable for the specified time point.
    End point type
    Secondary
    End point timeframe
    Pre-dose and at 0.25, 0.5, 1, 2, 4 and 12 hours post-dose on Day 1 and Week 5
    End point values
    		 VX-147
    Number of subjects analysed
    16
    Units: micrograms per milliliter (mcg/ml)
    arithmetic mean (standard deviation)
        15 mg qd: Day 1 (n=15)
    0.168 ( 0.0630 )
        45 mg qd: Week 5 (n=14)
    0.846 ( 0.303 )
    No statistical analyses for this end point

    Secondary: Observed Pre-dose Concentration (Ctrough) of VX-147

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    End point title
    		 Observed Pre-dose Concentration (Ctrough) of VX-147
    End point description
    PK set included all subjects who have received at least 1 dose of study drug in the treatment period. Here, the "n" signifies subjects who were evaluable for the specified time point.
    End point type
    Secondary
    End point timeframe
    Pre-dose and on Day 8, 15, Week 3, 5, 9 and 13
    End point values
    		 VX-147
    Number of subjects analysed
    16
    Units: mcg/ml
    arithmetic mean (standard deviation)
        15 mg qd: Day 8 (n=16)
    0.153 ( 0.0984 )
        15 mg qd: Day 15 (n=15)
    0.123 ( 0.0796 )
        45 mg qd: Week 3 (n=15)
    0.379 ( 0.249 )
        45 mg qd: Week 5 (n=15)
    0.492 ( 0.265 )
        45 mg qd: Week 9 (n=14)
    0.446 ( 0.342 )
        45 mg qd: Week 13 (n=13)
    0.529 ( 0.302 )
    No statistical analyses for this end point

    Secondary: Area Under the Concentration Time-curve From 0 to 24 hours (AUC0-24hr) of VX-147

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    End point title
    		 Area Under the Concentration Time-curve From 0 to 24 hours (AUC0-24hr) of VX-147
    End point description
    PK set included all subjects who have received at least 1 dose of study drug in the treatment period. Here, the "n" signifies subjects who were evaluable for the specified time point.
    End point type
    Secondary
    End point timeframe
    Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5
    End point values
    		 VX-147
    Number of subjects analysed
    13
    Units: micrograms per hour milliliter(mcg-h/ml)
        arithmetic mean (standard deviation)
    15.3 ( 6.37 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 up to Week 17
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.1
    Reporting groups
    Reporting group title
    VX-147: Cohort 1
    Reporting group description
    		 Cohort 1 included subjects with urine protein to creatinine ratio (UPCR) approximately ≥3 g/g (± 10%) and <10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%).

    Reporting group title
    VX-147: Cohort 2
    Reporting group description
    		 Cohort 2 included subjects with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2.

    Reporting group title
    VX-147: Total
    Reporting group description
    		 All subjects received VX-147 at a dosage of 15 mg qd for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included subjects with urine protein to creatinine ratio (UPCR) approximately ≥3 g/g (± 10%) and <10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included subjects with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2.

    Serious adverse events
    		 VX-147: Cohort 1 VX-147: Cohort 2 VX-147: Total
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Uterine leiomyoma
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Deep vein thrombosis
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    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
    		 VX-147: Cohort 1 VX-147: Cohort 2 VX-147: Total
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 3 (100.00%)
    12 / 13 (92.31%)
    15 / 16 (93.75%)
    Investigations
    Blood bicarbonate decreased
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 13 (15.38%)
    2 / 16 (12.50%)
         occurrences all number
    0
    3
    3
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Electrocardiogram ST segment depression
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 13 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    1
    0
    1
    Transaminases increased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Injury, poisoning and procedural complications
    Vaccination complication
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 3 (33.33%)
    3 / 13 (23.08%)
    4 / 16 (25.00%)
         occurrences all number
    1
    4
    5
    Dizziness
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 13 (15.38%)
    2 / 16 (12.50%)
         occurrences all number
    0
    2
    2
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 13 (15.38%)
    2 / 16 (12.50%)
         occurrences all number
    0
    2
    2
    Peripheral swelling
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 13 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    1
    0
    1
    Abdominal pain lower
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Abdominal distension
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    2
    2
    Dyspepsia
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 13 (15.38%)
    2 / 16 (12.50%)
         occurrences all number
    0
    3
    3
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Diarrhoea
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 13 (7.69%)
    2 / 16 (12.50%)
         occurrences all number
    1
    1
    2
    Vomiting
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 13 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    1
    0
    1
    Nausea
         subjects affected / exposed
    1 / 3 (33.33%)
    2 / 13 (15.38%)
    3 / 16 (18.75%)
         occurrences all number
    1
    2
    3
    Respiratory, thoracic and mediastinal disorders
    Epistaxis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 13 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    1
    0
    1
    Skin and subcutaneous tissue disorders
    Decubitus ulcer
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 13 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    1
    0
    1
    Eczema
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Dry skin
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Renal and urinary disorders
    Pollakiuria
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Psychiatric disorders
    Thinking abnormal
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Depressed mood
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Back pain
         subjects affected / exposed
    0 / 3 (0.00%)
    3 / 13 (23.08%)
    3 / 16 (18.75%)
         occurrences all number
    0
    3
    3
    Musculoskeletal chest pain
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Pain in extremity
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Synovitis
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Infections and infestations
    COVID-19
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Tinea pedis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 13 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    1
    0
    1
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Urinary tract infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Tooth abscess
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Vulvovaginal mycotic infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Mar 2020
    Amended to specify the doses to be used, added optional Cohort 2 and modified the schedule of assessments and eligibility criteria.
    23 Sep 2020
    Amended the eligibility criteria.
    11 Dec 2020
    Amended the eligibility criteria and also specified that both cohorts will be analyzed for the primary endpoint.
    09 Apr 2021
    Amended the eligibility criteria, updated the study restrictions, and contraceptive requirements.
    26 Jul 2021
    Amended to specify that Cohort 1 will enroll “up to 10 subjects” to clarify that enrollment could be stopped before 10 subjects were enrolled.

    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
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