Extrait

Background and study aims This study aims to evaluate a new ointment to treat an inherited skin tumour condition called cylindromatosis. Patients from families with this condition carry an error in their DNA (CYLD mutation) that results in the development of multiple skin tumours on the face, scalp and trunk. These 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 in laboratory tests blocking it with drugs called TRK inhibitors results in the tumour cells dying. We propose a study 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. Who can participate? Patients aged 18 and over from families with known CYLD mutations, who are scheduled to have a tumour removed. What does the study involve? This is a two-part study. In part 1 participants are provided with active CT327 ointment and a spatula to help them to apply the correct amount of ointment. The dose is one application (two spatulas) in the evening to the selected tumour (scheduled for removal) as directed. The aim of part 1 is to determine the safety of CT327 in CYLD mutation carriers. The number of patients who experience severe treated skin site reactions is measured over a 4-week period. In part 2 participants are randomly allocated to one of two groups and provided with active CT327 and placebo (dummy) ointment. One group applies the active ointment to tumours on the right side of their body and placebo ointment to the left side. The other group applies the placebo ointment to tumours on the right side of their body and active ointment to the left side. The dose is one application (one spatula) in the evening to each selected tumour (4-5 on each side) as directed. The number of tumours responding to treatment is measured after 12 weeks. What are the possible benefits and risks of participating? Not provided at time of registration Where is the study run from? Royal Victoria Infirmary (UK) When is the study starting and how long is it expected to run for? October 2014 to November 2016 Who is funding the study? Wellcome Trust (UK) Who is the main contact? Amy Cranston [email protected]

Critère d'inclusion

• Topic: Cancer, Genetics, Dermatology; Subtopic: Head and Neck Cancer, Genetics Research and Congenital Disorders (all subtopics), Skin (all Subtopics); Disease: Head and Neck, Genetics Research and Congenital Disorders, Dermatology

Liens

• ictrp
• isrctn