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    Summary
    EudraCT Number:2007-002657-23
    Sponsor's Protocol Code Number:VX06-770-101
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2007-07-09
    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 ConcernedGermany - BfArM
    A.2EudraCT number2007-002657-23
    A.3Full title of the trial
    A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study of VX-770 to Evaluate Safety, Pharmacokinetics, and Biomarkers of CFTR Activity in Cystic Fibrosis (CF) Subjects with Genotype G551D
    A.4.1Sponsor's protocol code numberVX06-770-101
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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-770
    D.3.2Product code VX-770, VRT-813077
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNNA
    D.3.9.2Current sponsor codeVX-770
    D.3.9.3Other descriptive nameVRT-813077
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 Information not present in EudraCT
    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 nameVX-770
    D.3.2Product code VX-770, VRT-813077
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNNA
    D.3.9.2Current sponsor codeVX-770
    D.3.9.3Other descriptive nameVRT-813077
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 Information not present in EudraCT
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    cystic fibrosis
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10011762
    E.1.2Term Cystic fibrosis
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - Evaluate the safety and tolerability of multiple dose administrations of orally administered VX-770 given to cystic fibrosis (CF) subjects with genotype G551D.
    E.2.2Secondary objectives of the trial
    - Examine biomarkers of CFTR activity following multiple dose administrations of orally administered VX-770 given to CF subjects with genotype G551D.
    - Investigate the pharmacokinetics (PK) of VX-770, following multiple dose administrations of orally administered VX-770 given to CF subjects with genotype G551D.
    - To select a dose of VX-770 for further study.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female with confirmed diagnosis of cystic fibrosis, and must be accompanied by either chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities and a positive sweat chloride value greater or equal to 60 mmol/L [by quantitative pilocarpine iontophoresis] or a sweat sodium greater or equal to 60 mmol/L on at least 1 occasion.
    2. In Part 1, must have the G551D mutation in at least 1 allele (any known or unknown mutations allowed in second allele with the exception of R117H or 2789 + 5G-->A, mutation) or, in Part 2, must have the G551D, R117H, or 2789 + 5G-->A mutation in at least 1 allele (any known or unknown mutations allowed in the second allele). The CF mutations must be appropriately documented in the medical record.
    3. Age 18 years or older.
    4. Weight 40 kg.
    5. Negative pregnancy test (for women of childbearing potential).
    6. Forced expiratory volume in 1 second being 40% of predicted normal for age, gender, and height (Hankinson standards): prebronchodilator value.
    7. Oxygen saturation (pulse oximetry) ³92% on room air.
    8. Hematology and clinical chemistry of blood and urine results with no clinically significant abnormalities that would interfere with the study assessments (as judged by the medical investigator).
    9. Able to understand and comply with protocol requirements, restrictions and instructions and likely to complete the study as planned.
    10. Willing to use at least 1 effective method of birth control (excluding hormonal contraceptives) during the study and for 90 days after the last dose of study drug. 11. Willing to remain on a stable medication regimen for the duration of study participation.
    E.4Principal exclusion criteria
    1. History of any illness 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 a history of cirrhosis; a history of relevant drug or food allergies; history of cardiovascular or central nervous system disease; history or presence of clinically significant pathology; or history of mental illness.
    2. Ongoing acute illness including acute respiratory or lower respiratory infections. Subjects should not have a pulmonary exacerbation (see Section 23 of protocol for definition) or changes in therapy for pulmonary disease within 14 days before the first dose of study drug.
    3. Pregnant, planning a pregnancy, or breast-feeding.
    4. Hemoglobin <10 gm/dL.
    5. Serum albumin <2.5 g/dL
    6. Abnormal liver function greater or equal to 3x upper limit of normal: (AST, ALT, GGT, ALP, total bilirubin).
    7. History of abnormal renal function (creatinine clearance < 50 mL/min [using Cockcroft-Gault equation]) in the past year.
    8. History of prolonged QT and/or QTcF (Fridericia's correction) interval (>450 msec).
    9. History of solid organ or hematological transplantation.
    10. History of alcohol abuse or drug addiction (including cannabis, cocaine and opiates) during the past year.
    11. Ongoing participation in another therapeutic clinical trial, or prior participation in an investigational drug study without appropriate washout (washout period should be discussed with the Vertex medical monitor on a case-by-case basis, but in no instance shall it be <5 half-lives of the previous investigational study drug).
    12. Illness within 14 days before receiving the first dose of study drug (“illness” is defined as a recent non-serious, non-acute condition, e.g., common cold).
    13. Intranasal medication changes within 14 days prior to start of dosing (including corticosteroids, cromolyn, phenylephrine, etc).
    14. Change of treatment with systemic antibiotics within 14 days prior to study drug dosing.
    15. Required use of continuous (24 hr/d) supplemental oxygen by nasal cannula.
    16. Concomitant use of any inhibitors or inducers of CYP3A4 including: acetazolamide, azole antifungals, clarithromycin, dalfopristin, diltiazem, erythromycin, fluoxetine, fluvoxamine, grapefruit juice, itraconazole, ketoconazole, voriconazole, loratadine, nefazadone, niacinamide, nicotinamide, propoxyphene, quinine, quinupristin, troleandomycin, valproate, verapamil, zileuton, felbamate, methsuximide, theophylline, and St. John’ s Wort.
    17. Not able to have Nasal potential differences performed due to physical or other constraints.
    E.5 End points
    E.5.1Primary end point(s)
    Primary endpoint:
    Safety and tolerability assessments based on clinical evaluations, laboratory assessments, and adverse events during administration of VX-770 for 14 or 28 days.

    Secondary endpoint:
    Nasal Potential Difference (NPD)
    Sweat Chloride
    Forced expiratory volume in 1 second
    VX-770 pharmacokinetic parameters
    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 Yes
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Part 1 is a cross over study, while part 2 is a parallel group study
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    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 No
    E.8.5The trial involves multiple Member States No
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    E.8.7Trial has a data monitoring committee Yes
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 36
    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)
    All study subjects, including drop-outs, will return to the clinic for a follow-up visit approximately 7 days after administration of the last dose of study drug to assess subjects' safety.
    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 Decision2007-10-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2007-06-25
    P. End of Trial
    P.End of Trial StatusCompleted
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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
    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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