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Topical kinase inhibition therapy for cutaneous cylindromas

Topical kinase inhibition therapy for cutaneous cylindromas

Research Summary:

This project aims to evaluate the ability of a new ointment to treat an inherited skin tumour condition. 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. These tumours (collectively called CYLD defective tumours) are disfiguring, can be painful, and may ulcerate and bleed. Surgery is the only available treatment, and up to 1 in 4 patients with this condition undergo removal of their entire scalp to manage this condition. We have recently discovered an abnormal signal in the tumour cells called TRK. This signal is recognised to give tumour cells the ability to survive, and blocking it with drugs called TRK inhibitors result in the tumour cells dying in laboratory tests. We propose a trial of an ointment form of TRK inhibitor as a means to reduce tumour growth in these patients. We have partnered with a drug company (Creabilis) who have already produced this ointment (called CT327) for trials in skin conditions. Should this be effective, it potentially could be used by patients in the future to treat early tumours and reduce the number of operations they would otherwise undergo.

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 crosscontamination
•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

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 or have recently changed (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 1 treated site will not be eligible
•Use of any other topically administered treatments at the treatment site

Study is still open to recruitment – scheduled to close 30/11/2016

Study Contact – Amy Cranston amy.cranston@ncl.ac.uk

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