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
- Conditions
- Patients with germline CYLD mutations (OMIM 605018). These include Brooke-Spiegler Syndrome, familial cylindromatosis and multiple familial trichoepitheliomas (OMIM #605041, #132700, #601606 respectively).Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
- Registration Number
- EUCTR2014-001342-21-GB
- Lead Sponsor
- ewcastle upon Tyne Hospitals NHS Foundation Trust
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 15
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
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 8
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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method