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    Summary
    EudraCT Number:2022-002746-40
    Sponsor's Protocol Code Number:VX22-864-108
    National Competent Authority:Ireland - HPRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2022-11-21
    Trial 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 ConcernedIreland - HPRA
    A.2EudraCT number2022-002746-40
    A.3Full title of the trial
    A Phase 2, Open-label Study Evaluating Efficacy and Safety of VX-864 in Subjects With Alpha-1 Antitrypsin Deficiency Who Have the PiZZ Genotype, Over 48 Weeks
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
    A.4.1Sponsor's protocol code numberVX22-864-108
    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 codeMA 02210-1862
    B.5.3.4CountryUnited States
    B.5.4Telephone number001 877 634 878
    B.5.5Fax number001 510 595 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 nameVX864
    D.3.2Product code VX864
    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.3.9.2Current sponsor codeVX-864
    D.3.9.3Other descriptive nameVX-864
    D.3.9.4EV Substance CodeSUB199437
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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.IMP: 2
    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 nameVX864
    D.3.2Product code VX864
    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.3.9.2Current sponsor codeVX-864
    D.3.9.3Other descriptive nameVX-864
    D.3.9.4EV Substance CodeSUB199437
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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
    Alpha 1 antitrypsin deficiency in Subjects With the PiZZ Genotype
    E.1.1.1Medical condition in easily understood language
    Alpha 1 antitrypsin deficiency
    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 23.1
    E.1.2Level PT
    E.1.2Classification code 10083869
    E.1.2Term Alpha-1 antitrypsin deficiency
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic 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 efficacy of VX-864 on blood levels of functional alpha-1 antitrypsin (AAT) in individuals with the PiZZ genotype
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of VX-864 on blood levels of antigenic AAT in individuals with the PiZZ genotype
    To evaluate the efficacy of VX-864 on blood levels of Z-polymer in individuals with PiZZ genotype
    To evaluate the efficacy of VX-864 on liver Z-polymer in individuals with the PiZZ genotype (in Group B only)
    To evaluate the safety and tolerability of VX-864 in individuals with the PiZZ genotype
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject will 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 (Section 11.6.8.1), and other study procedures.
    3. In subjects not undergoing liver biopsies (Group A):
    • Subjects between the ages of 18 and 80 years, inclusive, at the time of ICF signing. In subjects undergoing liver biopsies (Group B):
    • Subjects between the ages of 18 and 70 years, inclusive at the time of the ICF signing.
    4. Subjects must have a PiZZ genotype confirmed during screening.
    • All subjects must have PiZZ genotype tested at the central laboratory, except for subjects who have had historical genotype results obtained via the central laboratory as part of another Vertex study.
    • A historical genotype (not from another Vertex study) may be used to establish eligibility with medical monitor approval. Subjects who have been enrolled, and whose screening genotype from the central laboratory does not confirm study eligibility, must be discontinued from the study.
    • In Group A subjects, the PiZZ genotype must be confirmed before Day 1 dosing. In Group B subjects, the PiZZ genotype must be confirmed prior to the pretreatment liver biopsy.
    5. In subjects not undergoing biopsies (Group A)
    • A forced expiratory volume in 1 second (FEV1) value ≥30% of predicted mean for age, sex, and height (equations of the Global Lung Function Initiative [GLI]) during screening or a historical result within 1 year before the initial Screening Visit. Post-bronchodilator spirometry measurements must meet American Thoracic Society (ATS)/European Respiratory Society (ERS) criteria for acceptability and repeatability.
    In subjects undergoing biopsies (Group B)
    • An FEV1 value ≥40% of predicted mean for age, sex, and height (equations of the GLI) during screening or a historical result within 1 year before the initial Screening Visit. Post-bronchodilator spirometry measurements must meet ATS/ ERS criteria for acceptability and repeatability.
    6. Body mass index (BMI) of 18.0 to 35 kg/m2, inclusive.
    7. Screening antigenic AAT <8 µM. For subjects who have received augmentation therapy within 42 days before the initial screening visit, results used to confirm eligibility must be drawn >42 days after the last dose of augmentation therapy. Levels of antigenic AAT must be obtained from the central laboratory and results confirmed in all subjects. In Group A subjects, it must be confirmed prior to Day 1. In Group B subjects, it must be confirmed prior to the pretreatment liver biopsy.
    E.4Principal exclusion criteria
    1. History of any illness or any clinical condition that, in the opinion of the investigator or the subject’s general practitioner, might confound the results of the study or pose an additional risk in administering study drug to the subject. This may include, but is not limited to, history of relevant drug or food allergies; history of cardiovascular or central nervous system disease; history or presence of clinically significant pathology; history of mental disease; and history of cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage 0 cervical carcinoma in situ (all 3 with no recurrence for the last 5 years).

    2. Solid organ or hematological transplantation or is currently on a transplant list.

    3. Subjects who have undergone gastrectomy or major resection of the small bowel or proximal colon.

    4. All clinically important pulmonary disease as deemed by investigator, including but not limited to unstable AATD‑related chronic obstructive pulmonary disease (COPD), interstitial lung disease, cystic fibrosis, pulmonary hypertension with or without cor pulmonale, history of pulmonary embolism, or malignant lung cancer.

    5. Subjects for whom discontinuation of augmentation therapy is not considered to be in their best interest, based on the clinical judgement of the treating physician.

    6. Only in subjects undergoing liver biopsies (in Group B), any contraindication to a percutaneous liver biopsy including a bleeding diathesis, ascites, or chronic need for treatment with anti-coagulants or anti-platelet agents.

    7. Current diagnosis of active hepatitis and/or significant chronic liver disease of any etiology other than AATD-associated non-cirrhotic liver disease including, but not limited to hepatic cirrhosis, portal hypertension, or confirmed or suspected esophageal varices.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in blood levels of functional AAT at Week 48
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 48
    E.5.2Secondary end point(s)
    • Change from baseline in blood levels of functional AAT over time
    • Change from baseline in blood levels of antigenic AAT over time
    • Change from baseline in blood levels of Z-polymer over time
    • Change from baseline in Z-polymer accumulation in the liver over time (in Group B only)
    • Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values, standard 12-lead ECGs, and vital signs
    E.5.2.1Timepoint(s) of evaluation of this end point
    From baseline to safety follow up
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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
    Germany
    Ireland
    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 years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days1
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days1
    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: 15
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 3
    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 Decision2023-01-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-04-11
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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