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    Summary
    EudraCT Number:2020-000185-42
    Sponsor's Protocol Code Number:VX19-147-101
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-05-26
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2020-000185-42
    A.3Full title of the trial
    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.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 2a Study of VX 147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis.
    A.4.1Sponsor's protocol code numberVX19-147-101
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorVertex Pharmaceuticals Incorporated
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportVertex Pharmaceuticals Incorporated
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVertex Pharmaceuticals Incorporated
    B.5.2Functional name of contact pointClinical Trials and Medical Info
    B.5.3 Address:
    B.5.3.1Street Address50 Northern Avenue
    B.5.3.2Town/ cityBoston
    B.5.3.3Post code02210-1862
    B.5.3.4CountryUnited States
    B.5.4Telephone number001877634 8789
    B.5.5Fax number001510595 8183
    B.5.6E-mailmedicalinfo@vrtx.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameVX-147
    D.3.2Product code VX-147
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVX147
    D.3.9.2Current sponsor codeVX-147
    D.3.9.3Other descriptive nameVX-147
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    APOL1-mediated Focal segmental glomerulosclerosis (FSGS)
    E.1.1.1Medical condition in easily understood language
    Focal segmental glomerulosclerosis
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10067757
    E.1.2Term Focal segmental glomerulosclerosis
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the ability of VX-147 to reduce proteinuria
    E.2.2Secondary objectives of the trial
    • To evaluate the safety and tolerability of VX-147
    • To characterize the PK of VX-147
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    The following criteria are applicable for Cohort 1 and Cohort 2:
    1. Willing to sign and date an informed consent form (ICF).
    2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines and other study procedures.
    3. An APOL1 genotype of G1/G1, G2/G2, or G1/G2 obtained with a Vertex-approved clinical study assay.
    4. Between the ages of 18 and 60 years, inclusive.
    5. Body mass index (BMI) of 18.0 to 40.0 kg/m2, inclusive, and a total body weight >50 kg.
    6. FSGS diagnosed by kidney biopsy, with the exception of the tip variant, as confirmed through the eligibility review process.
    7. Estimated glomerular filtration rate (eGFR) ≥45 mL/min/1.73 m2 based on the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation.
    The following criteria are applicable for Cohort 1:
    8. A UPCR ratio of ≥3 g/g and <10 g/g in the first morning void on 3 measurements collected on at least 3 separate days within a 7-day period, during the Screening Period. All 3 measurements must meet this criterion.
    9. There should be no plan to start, stop, or modify dosing for an angiotensin converting enzyme (ACE) inhibitor, angiotensin receptor blocker (ARB), neprilysin inhibitor, sodium-glucose co-transporter-2 inhibitor, or renin inhibitor during the Treatment Period or Follow-up Period.
    The following criteria are applicable for Cohort 2:
    10. A UPCR ratio of ≥ 2 g/g and <10 g/g in the first morning void on 3 measurements collected on at least 3 separate days within a 7-day period, during the Screening Period. All 3 measurements must meet this criterion.
    11. There should be no plan to start, stop, or modify dosing for an ACE inhibitor, ARB, neprilysin inhibitor, sodium-glucose co-transporter-2 inhibitor, renin inhibitor, or systemic corticosteroids during the Treatment Period or Follow-up Period.
    E.4Principal exclusion criteria
    The following criteria are applicable for Cohort 1 and Cohort 2:
    1. History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug(s) to the subject. This includes, but is not limited to, the following:
    • Solid organ or bone marrow transplantation
    • Cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage 0 cervical carcinoma in situ (each being disease-free for the last 5 years)
    • Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator
    • Clinically significant liver disease
    • Ongoing alcohol or drug abuse as determined by the investigator
    • Any condition possibly affecting drug absorption (e.g., gastrectomy, gastrointestinal tract surgery except appendectomy and cholecystectomy)
    • Stroke or myocardial infarction within 6 months before Day 1
    2. Evidence of non-APOL1-mediated FSGS. This includes but is not limited to the following:
    • FSGS occurring concomitantly to administration of drugs known to induce FSGS, including but not limited to lithium, interferon, and bisphosphonates (e.g., pamidronate), or FSGS occurring in a subject using intravenous illicit drugs at the time of diagnosis.
    • Evidence of another underlying kidney disease that can cause FSGS, including evidence of congenital anomaly of the kidney or urinary tract (CAKUT) on renal ultrasound, history of CAKUT, history of nephrectomy.
    • FSGS occurring in a subject with known sickle cell disease.
    • Known genetic mutation other than APOL1 G1 or G2 that is associated with FSGS.
    • Positive serology for human immunodeficiency virus-1 (HIV-1) or human immunodeficiency virus-2 (HIV-2).
    3. Evidence of kidney disease other than FSGS on kidney biopsy, as assessed by the eligibility review process.
    4. Kidney biopsy showing the tip variant of FSGS, as assessed by the eligibility review process.
    5. Any of the following abnormal laboratory values at screening:
    • Serum albumin <1 g/dL
    • Total bilirubin ≥1.5 × upper limit of normal (ULN)
    • Aspartate transaminase (AST) or alanine transaminase (ALT) ≥2 × ULN
    • Potassium above the ULN
    • Hemoglobin <10 g/dL.
    6. Positive for hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) antibody during screening.
    7. Risk factors for Torsade de Pointes (e.g., familial long QT syndrome, chronic hypokalemia, heart failure) or concomitant medications that prolong the QT/QTc interval or any history of cardiac disorders that, in the opinion of the investigator, might put the subject at risk or may confound the results of the study.
    8. Any clinically significant ECG abnormality (as determined by the investigator) or median QTcF of triplicate standard 12 ECGs >450 msec at screening.
    9. Screening blood pressure ≥160 mm Hg (systolic) or ≥100 mm Hg (diastolic), based on the average of 3 measurements.
    10. Pregnant or nursing female subjects.
    11. Subjects of reproductive potential who are not willing to follow the contraception requirements outlined in Section 11.6.5.1.
    12. Plan to travel to countries where sleeping sickness is endemic, from the Screening Visit through 1 week after the last dose of study drug.
    13. Subject, or close relative of the subject, is the investigator or a subinvestigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site.
    14. Hypersensitivity to investigational medicinal product or to any of its excipients.
    The following criterion is applicable for Cohort 1
    15. Use of the substances or activities as indicated in Section 9.5 during the specified times, including but not limited to ongoing treatment with corticosteroids (any dose level) or another immunosuppressive drug.
    The following criterion is applicable for Cohort 2
    16. Use of the substances or activities as indicated in Section 9.5 during the specified times, including but not limited to ongoing treatment with high doses of corticosteroids (>10 mg/day of prednisone or prednisone equivalent) or another immunosuppressive drug.

    E.5 End points
    E.5.1Primary end point(s)
    Percent change from baseline in urine protein to creatinine ratio (UPCR) at Week 13
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline to Week 13
    E.5.2Secondary end point(s)
    Secondary End point 1- Safety and tolerability based on adverse events (AEs), clinical laboratory values (i.e., hematology, serum chemistry, urinalysis, coagulation studies), standard 12-lead ECGs, and vital signs

    secondary Point 2- Plasma PK of VX-147
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary Endpoint 1: Continuous from signing of ICF or electronic ICF through Safety Follow-up Visit

    Secondary Endpoint 2: From Baseline to Week 13 and ETT
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    France
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months12
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 4
    F.4.2.2In the whole clinical trial 20
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-05-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-06-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-01-25
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