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    Summary
    EudraCT Number:2014-001342-21
    Sponsor's Protocol Code Number:NCTUTRK1(6840)
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-09-08
    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 ConcernedUK - MHRA
    A.2EudraCT number2014-001342-21
    A.3Full title of the trial
    Topical tropomysin kinase (TRK) inhibitor as a treatment for inherited CYLD defective skin tumours (TRAC)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A trial to find out if CT327 ointment (a TRK inhibitor) is safe in patients with inherited CYLD defective skin tumours and if the tumours respond to the treatment
    A.3.2Name or abbreviated title of the trial where available
    Tropomyosin Receptor kinase Antagonism in Cylindromatosis (TRAC)
    A.4.1Sponsor's protocol code numberNCTUTRK1(6840)
    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 SponsorNewcastle upon Tyne Hospitals NHS Foundation Trust
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCreabilis
    B.4.2CountryUnited Kingdom
    B.4.1Name of organisation providing supportWellcome Trust
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNewcastle University
    B.5.2Functional name of contact pointNeil Rajan
    B.5.3 Address:
    B.5.3.1Street AddressInternational Centre for Life, Central Parkway
    B.5.3.2Town/ cityNewcastle upon Tyne
    B.5.3.3Post codeNE1 3BZ
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number01912418813
    B.5.6E-mailneil.rajan@ncl.ac.uk
    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 nameCT327
    D.3.2Product code CT327
    D.3.4Pharmaceutical form Ointment
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPTopical use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCT327
    D.3.9.4EV Substance CodeAS2
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    D.3.10.2Concentration typeequal
    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 placeboOintment
    D.8.4Route of administration of the placeboTopical use (Noncurrent)
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients with germline CYLD mutations (OMIM 605018). These include Brooke-Spiegler Syndrome, familial cylindromatosis and multiple familial trichoepitheliomas (OMIM #605041, #132700, #601606 respectively).
    E.1.1.1Medical condition in easily understood language
    Patients in families with this condition carry an error in their DNA that results in the development of multiple skin tumours on the face, scalp and trunk.
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The principal research objective for Cohort 1 is to find out whether CT327 (an ointment with a drug called a TRK inhibitor in it) is a safe treatment for patients with CYLD defective tumours (lumps). The principal research objective for Cohort 2 is to investigate whether CYLD defective tumours (lumps) respond to CT327.
    E.2.2Secondary objectives of the trial
    Secondary objectives for Cohort 1 are to measure quality of life (questionnaire), skin and tumour (lump) appearance and patient treatment compliance (patient diary). Secondary objectives for Cohort 2 are to establish the way that the study drug works on the size of CYLD defective tumours (lumps) using genetic techniques and protein measurement.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Cohort 1:
    •Males and females age 18 years and older
    •Patients from genotyped pedigrees with known CYLD mutations; or if they have a clinical phenotype compatible with this diagnosis
    •Patients that are suitable for the trial will have at least one eligible tumour
    •The eligible tumour will be scheduled for removal >4 weeks from consent
    •The eligible tumour must be no more than 3cm in size
    •For women of childbearing age: a negative pregnancy test is required prior to study entry, and on completion of trial treatment. The patient must be using an adequate contraception method and agree to continue using this throughout the trial and for at least 2 weeks after stopping trial medication
    •Sexually active men must agree to use barrier forms of contraception
    •The recruiting clinician must be confident that the patient understands the consent process and has the capacity and willingness to provide fully informed consent for participation in the trial

    Cohort 2:
    •Males and females age 18 years and older
    •For women of child bearing age: a negative pregnancy test is required prior to study entry, and on completion of trial treatment. The patient must be using an adequate contraception method and agree to continue using this throughout the trial and for at least 2 weeks after stopping trial medication
    •Patients from genotyped pedigrees with known CYLD mutations, or if they have a clinical phenotype compatible with this diagnosis
    •Patients will optimally have 8-10 eligible tumours
    •Eligible tumours will be less than 1 cm in diameter and no more than 2 cm in diameter at the base
    •Eligible tumours must be spaced at least 1 cm apart from other eligible tumours to avoid cross-contamination
    •The recruiting clinician must be confident that the patient understands the consent process and has the capacity and willingness to provide fully informed consent for participation in the trial
    •Patients who have completed Phase 1b without adverse reaction and after completing a minimum 2 week treatment free washout period
    E.4Principal exclusion criteria
    Cohort 1:
    •Patients aged <18 years
    •Patients without CYLD defective tumours
    •CYLD defective tumours which are ulcerated (these tumours will be managed according to standard practice of care)
    •The eligible tumour is due to be removed <4 weeks from consent
    •Pregnancy or lactation
    •Women of childbearing age and sexually active men whom do not wish to use contraception whilst on the study
    •Severe incapacity of higher function such that fully informed consent cannot be achieved, to be determined by clinical judgement
    •Use of any other topically administered treatments at the treatment site

    Cohort 2:
    •Patients aged <18 years
    •Patients without multiple CYLD defective tumours
    •Pregnancy or lactation
    •Women of childbearing age and sexually active men whom do not wish to use contraception whilst on the study
    •CYLD defective tumours which are ulcerated, have recently changed or are painful (these tumours will be managed according to standard practice of care)
    •Severe incapacity of higher function such that fully informed consent cannot be achieved, to be determined by clinical judgement
    •Significant concurrent illness
    •Patients who developed an adverse reaction to CT327 in cohort 1(score of 4 or above on the modified Draize score)
    •Patients who have taken part in cohort 1and not completed a minimum 2 week treatment free washout period
    •Large tumours >2cm base diameter will not be eligible
    •Any tumour within 10cm of an excision scar of a cohort 1treated site will not be eligible
    •Use of any other topically administered treatments at the treatment site
    E.5 End points
    E.5.1Primary end point(s)
    The primary outcome measure for Cohort 1 is the number of patients with severe treated skin site reactions as determined by Modified Draize score, after a 4 week treatment period. The primary outcome measure for Cohort 2 is the proportion of tumours responding to treatment by 12 weeks.
    E.5.1.1Timepoint(s) of evaluation of this end point
    In Cohort 1 the timepoint of evaluation is at 4 weeks. In Cohort 2 the timepoints of evaluation are at 4 weeks and 12 weeks.
    E.5.2Secondary end point(s)
    In Cohort 1 the secondary endpoints are; Impact of disease on quality of life, acceptability of treatment, and concentration of CT327 in plasma.

    In Cohort 2 the secondary endpoints are; the number of adverse events within a planned 12 week treatment period, expression of targets of TRK signalling in tumour biopsies as determined by QPCR and immunohistochemistry, concentration of CT327 in plasma and a proportion of tissue samples.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The timepoint of evaluation in cohort 1 is at 4 weeks.

    The timepoints of evaluation in cohort 2 is at 4 weeks and 12 weeks.
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Trial of treatment safety and efficacy.
    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 Yes
    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 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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    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.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 8
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state28
    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)
    Due to the early phase of this trial there are no arrangements to continue treatment past the 3 months of treatment in cohort 2.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation North East and North Cumbria Local Clinical Research Network (NENC LCRN)
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-09-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-09-04
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-07-31
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