Topic: Cancer, Genetics, Dermatology
Conditions
Interventions
Cohort 1: active trial medication containing CT327 at 0.5%w/w will be provided as ointment in 20 g glass jars. Patients will be provided with a spatula to help them to apply the correct amount of oint
Sponsors
Newcastle upon Tyne Hospitals NHS Foundation Trust (UK)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Cohort 1: 1. Males and females age 18 years and older 2. Patients from genotyped pedigrees with known CYLD mutations; or if they have a clinical phenotype compatible with this diagnosis 3. Patients that are suitable for the trial will have at least one eligible tumour 4. The eligible tumour will be scheduled for removal >4 weeks from consent 5. The eligible tumour must be no more than 3cm in size 6. 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 7. Sexually active men must agree to use barrier forms of contraception 8. 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: 1. Males and females age 18 years and older 2. 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 3. Patients from genotyped pedigrees with known CYLD mutations, or if they have a clinical phenotype compatible with this diagnosis 4. Patients will optimally have 8-10 eligible tumours 5. Eligible tumours will be less than 1 cm in diameter and no more than 2 cm in diameter at the base 6. Eligible tumours must be spaced at least 1 cm apart from other eligible tumours to avoid crosscontamination 7. 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 8. Patients who have completed Phase 1b without adverse reaction and after completing a minimum 2 week treatment free washout period
Exclusion criteria
Exclusion criteria: Cohort 1: 1. Patients aged 2cm base diameter will not be eligible 11. Any tumour within 10cm of an excision scar of a cohort 1 treated site will not be eligible 12. Use of any other topically administered treatments at the treatment site
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Cohort 1: Number of patients with severe treated skin site reactions as determined by Modified Draize score. Cohort 2: The proportion of tumours responding to treatment by 12 weeks. | — |
Secondary
| Measure | Time frame |
|---|---|
| Cohort 1: 1. Patient reported quality of life using patient reported QoL tools (EQ5D, DLQI) 2. Acceptability of treatment according to patient treatment questionnaire 3. Adverse events within a planned 4-week treatment period 4. Compliance including reasons for non-compliance Cohort 2: 1. Change in tumour volume from baseline (pre-randomisation) to 12 weeks 2. Adverse events within a planned 12-week treatment period 3. Compliance including reasons for non-compliance 4. Confirmation of the definition of response (currently according to WHO RECIST criteria where response is defined as >30% reduction in tumour volume, this will be used as a benchmark) 5. Expression of targets of TRK signalling in tumour biopsies as determined by QPCR and immunohistochemistry 6. Patient reported quality of life using patient reported QoL tools (EQ5D, DLQI) 7. Assessment of acceptability of trial treatment according to patient treatment questionnaire | — |
Countries
United Kingdom
Outcome results
None listed