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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
    		 v1
    This version publication date
    25 Dec 2022
    First version publication date
    25 Dec 2022
    Other versions
    		 v2

    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
    United Kingdom: 3
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    United States: 11
    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) and Part B (Optional Exploratory Off-treatment Follow-up Period). The primary and secondary efficacy 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
    		 Cohort 1: Subjects received VX-147 dosage once daily (qd) for 2 weeks in the treatment period based on a range of urine protein to creatinine ratio (UPCR) values greater than or equal to (≥) 3 g/g (± 10%) and less than(<) 10 g/g at screening.Cohort 2: Subjects received VX-147 dosage once daily for 13 weeks in the treatment period based on a range of UPCR values ≥0.8 g/g (± 10%) and <2.7 g/g at screening.
    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
    		 Cohort 1: Subjects received VX-147 dosage once daily (qd) for 2 weeks in the treatment period based on a range of urine protein to creatinine ratio (UPCR) values greater than or equal to (≥) 3 g/g (± 10%) and less than(<) 10 g/g at screening.Cohort 2: Subjects received VX-147 dosage once daily for 13 weeks in the treatment period based on a range of UPCR values ≥0.8 g/g (± 10%) and <2.7 g/g at screening.

    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

    End points

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    End points reporting groups
    Reporting group title
    VX-147
    Reporting group description
    		 Cohort 1: Subjects received VX-147 dosage once daily (qd) for 2 weeks in the treatment period based on a range of urine protein to creatinine ratio (UPCR) values greater than or equal to (≥) 3 g/g (± 10%) and less than(<) 10 g/g at screening.Cohort 2: Subjects received VX-147 dosage once daily for 13 weeks in the treatment period based on a range of UPCR values ≥0.8 g/g (± 10%) and <2.7 g/g at screening.

    Primary: Part A : Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR)

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    End point title
    		 Part A : Percent Change From Baseline in Urine Protein to Creatinine Ratio (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

    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
    		 Subjects received VX-147 dosage once daily (qd) for 2 weeks in the treatment period based on a range of UPCR ≥3 g/g (± 10%) and <10 g/g at screening.

    Reporting group title
    VX-147: Cohort 2
    Reporting group description
    		 Subjects received VX-147 dosage once daily for 13 weeks in the treatment period based on a range of UPCR ≥ 0.8 g/g (± 10%) and <2.7 g/g at screening.

    Reporting group title
    VX-147: Total
    Reporting group description
    		 Subjects received VX-147 dosage qd for 2 weeks in the treatment period based on a range of UPCR values ≥3 g/g (± 10%) and <10 g/g at screening of Cohort 1. Subjects received VX-147 dosage once daily for 13 weeks in the treatment period based on a range of UPCR values ≥0.8 g/g (± 10%) and <2.7 g/g at screening of Cohort 2.

    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
    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
    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
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    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
    Dizziness
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 13 (15.38%)
    2 / 16 (12.50%)
         occurrences all number
    0
    2
    2
    Headache
         subjects affected / exposed
    1 / 3 (33.33%)
    3 / 13 (23.08%)
    4 / 16 (25.00%)
         occurrences all number
    1
    4
    5
    General disorders and administration site conditions
    Peripheral swelling
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Fatigue
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 13 (15.38%)
    2 / 16 (12.50%)
         occurrences all number
    0
    2
    2
    Gastrointestinal disorders
    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 pain
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 13 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    1
    0
    1
    Abdominal distension
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    2
    2
    Diarrhoea
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 13 (7.69%)
    2 / 16 (12.50%)
         occurrences all number
    1
    1
    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
    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
    Depressed mood
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Thinking abnormal
         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
    Pain in extremity
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 13 (7.69%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    1
    Musculoskeletal chest pain
         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
    Tooth abscess
         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
    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
    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.

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