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    Summary
    EudraCT Number:2017-001042-10
    Sponsor's Protocol Code Number:VX16-150-102
    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:2017-06-06
    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 number2017-001042-10
    A.3Full title of the trial
    A Phase 2, Randomized, Double-blind, Placebo-controlled, 6-Week, Parallel-design Study of the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate how safe and able to produce an effect in the body study drug VX-150 is in patients with pain that typically begins in the feet or hand
    A.4.1Sponsor's protocol code numberVX16-150-102
    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, Massachusetts
    B.5.3.3Post code02210
    B.5.3.4CountryUnited States
    B.5.4Telephone number +1 877 634-8789
    B.5.5Fax number+1 510 595-8183
    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 nameVX-150
    D.3.2Product code VX-150
    D.3.4Pharmaceutical form Capsule
    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 INN-
    D.3.9.2Current sponsor codeVX-150
    D.3.9.3Other descriptive nameVRT-1268150, VXc-150
    D.3.9.4EV Substance CodeSUB181412
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule
    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
    Pain Caused by Small Fiber Neuropathy
    E.1.1.1Medical condition in easily understood language
    Severe pain attacks that typically begin in the feet or hands
    E.1.1.2Therapeutic area Body processes [G] - Bones and nerves physological processes [G11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10033371
    E.1.2Term Pain
    E.1.2System Organ Class 10018065 - General disorders and administration site conditions
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10073937
    E.1.2Term Small fiber neuropathy
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of VX-150 for the treatment of pain caused by small fiber neuropathy
    E.2.2Secondary objectives of the trial
    • To evaluate the safety and tolerability of VX-150

    • To evaluate the PK of VRT-1207355 and the metabolite VRT-1268114
    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, and other study procedures

    3. Subjects (male and female) will be between the ages of 18 and 80 years, inclusive.

    4. Body mass index (BMI) of ≥18.0 kg/m2

    5. Diagnosis of small fiber neuropathy, as per European Federation Neurological Societies (EFNS)/American Academy of Neurology (AAN) guidelines, with pain for at least 3 months prior to screening

    6. Reduction below the 5th percentile of sex and age-adjusted normal values in epidermal nerve fiber density on punch skin biopsy at the distal site of the leg performed at or within 6 months of screening

    7. Presence of sural nerve responses

    8. Average NRS score between ≥4 and ≤9 reported in the daily diary on Days -7 through -1
    E.4Principal exclusion criteria
    1. History in the past 10 years of malignancy 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. Exposure to neurotoxic drugs (i.e., chemotherapy) since diagnosis of small fiber neuropathy. Untreated or uncontrolled connective tissue disorders, sarcoidosis, Sjögren’s syndrome, amyloidosis, Fabry’s disease, celiac disease, Lyme disease, autoimmune disorders (i.e., as assessed by anti-nuclear antibodies, rheumatoid factor, sedimentation rate, and/or lupus anticoagulant)
    including myasthenia gravis and Guillain-Barre syndrome, which in the opinion of the investigator makes the subject unsuitable for inclusion in this study.

    3. A known or clinically suspected infection with human immunodeficiency virus or hepatitis B or C viruses

    4. Current clinically significant liver or kidney dysfunction

    5. Current uncontrolled thyroid dysfunction

    6. Subjects with a diagnosis of diabetes who have any 1 of the following criteria:
     - HbA1c ≥11% at screening
    - are not stabilized on oral hypoglycemics and/or subcutaneous insulin or diet, in the opinion of the investigator
     - evidence of ulcers or severe nephropathy resulting from their diabetes
     - advanced retinopathy, defined as greater than State 3 (moderate non-proliferative diabetic retinopathy)
     - history of a clinical atherosclerotic event, such as myocardial infarction or stroke

    7. History of cardiac dysrhythmias requiring anti-arrhythmia treatment(s); or history or evidence of abnormal ECGs that in the opinion of the investigator or medical monitor would preclude the subject's participation in the study

    8. Standard 12-lead ECG demonstrating QTc >450 msec at screening. If QTc exceeds 450 msec, the ECG will be repeated 2 more times, and the average of the 3 QTc values will be used to determine the subject’s eligibility.

    9. Concomitant severe pain conditions (i.e., low back pain, radiculopathy, severe bone and musculoskeletal disorders) which may impair self-assessment of pain due to small fiber neuropathy

    10. Abnormal laboratory results indicative of any significant medical disease that, in the opinion of the investigator, would preclude the subject's participation in the study

    11. Other serious, acute, or chronic medical or psychiatric illness that, in the judgment of the investigator, could compromise subject safety, limit the subject’s ability to complete the study and/or compromise the objectives of the study

    12. Female subjects who are pregnant, nursing, or planning to become pregnant during the study or within 90 days after the last study drug dose

    13. Male subjects with a female partner who is pregnant, nursing, or planning to become pregnant during the study or within 90 days after the last study drug dose

    14. Use of restricted medication or food within the specified duration before the first dose of study drug

    15. Alcohol, analgesic/opioid, and/or illicit drug abuse as defined by the American Psychiatric Association Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, in the last 12 months before screening, or a positive test for drugs of abuse at screening
    • A positive drug screen for a known concomitant medication that is not otherwise exclusionary (e.g., benzodiazepines) will not disqualify subjects; however, marijuana and marijuana derivatives will not be allowed

    16. 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
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in the weekly average of daily pain intensity on the 11-point numeric rating scale (NRS), as reported in the daily diary, at Week 6
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 6
    E.5.2Secondary end point(s)
    • Proportion of subjects with ≥30% reduction in the weekly average of daily pain intensity on the 11-point NRS, as reported in the daily diary, at Week 6

    • Proportion of subjects with ≥50% reduction in the weekly average of daily pain intensity on the 11-point NRS, as reported in the daily diary, at Week 6

    • Change from baseline in the Daily Sleep Interference Scale (DSIS) at Week 6

    • Proportion of subjects categorized as improved at Week 6 on the patient global impression of change (PGIC) assessment

    • Change from baseline in pain intensity on the 11-point NRS, as reported at study visits, at Week 6

    • Plasma PK parameters of VRT-1207355 and the metabolite VRT-1268114

    • Safety and tolerability based on the Columbia Suicide Severity Rating Scale (C-SSRS), incidence and type of AEs, changes from baseline in clinically significant laboratory test results, vital signs, and ECGs at each visit
    E.5.2.1Timepoint(s) of evaluation of this end point
    • At week 6

    • Plasma PK parameters at day 7, week 3, week 6 and early termination visit

    • Safety and tolerability at each visit
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    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 Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA4
    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
    Canada
    Germany
    Italy
    Netherlands
    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 months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months7
    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: 86
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 28
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 114
    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)
    Standard of care
    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 Decision2017-07-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-01-17
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-11-08
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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