Study of Patidegib Gel 2%, 4%, and Vehicle in Gorlin Syndrome Patients
Research type
Research Study
Full title
Double- Blind, Randomized, Vehicle-Controlled Proof of Concept Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients
IRAS ID
195362
Contact name
John Lear
Contact email
Sponsor organisation
Blue Reg Pharma Consult (Blue Reg)
Eudract number
2015-004274-15
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
Study of Patidegib Gel 2%, 4%, and Vehicle in Gorlin Syndrome Patients
Patidegib is an investigational semi-synthetic small molecule, this topical drug product is generally safe to be tested in patients affected by the basal cell nevus syndrome (BCNS) (Gorlin syndrome, nevoid basal cell carcinoma syndrome; a rare autosomal dominant inherited disorder, that dramatically increases the risk of developing basal cell carcinoma BCCs as well as an increased risk of developing certain extra cutaneous tumours (e.g. medulloblastomas and rhabdomyosarcomas).
The purpose of the study is to assess:
1. The clinical efficacy of patidegib as defined by the percentage of decrease in greatest diameter of baseline treatment targeted Surgically Eligible basal cell carcinomas at given time points.
2. The molecular efficacy of treatment as defined by reduction in the hedgehog (HH) signalling pathway target gene GLI1 after treatment with patidegib gel 2% or 4% or vehicle applied twice daily for 6 weeks to treatment-targeted SEBs.
3. The safety and tolerability of treatment with patidegib gel 2% or 4% or vehicle applied twice daily for 26 weeks.The goal of the present trial is to evaluate topical patidegib’s safety, tolerability, and effects on the size of pre-existing SEBs and on the development of new SEBs. If the efficacy with topical patidegib can approach the level of efficacy of oral HH inhibitors seen in BCNS patients while avoiding their systemic side effects, it would represent a major advance for BCNS patients.
The drug is being developed in the hope to decrease the number of facial SEBs in BCNS patients by (i) shrinking pre-existing tumours and (ii) preventing the development of new tumours, by applying a topical HH inhibitor (patidegib) to produce good local effects without producing the side effects that occur with systemic administration of this class of drugs.
This is a multicentre, double-blind, randomised, vehicle-controlled study designed to
compare the efficacy and safety of patidegib gel, 2% and 4% in comparison with that of
vehicle. Approximately 18 adult patients will be enrolled and randomised in the study, with a 1:1:1 randomisation ratio, at 2 investigational centres in Europe.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
16/NW/0071
Date of REC Opinion
22 Feb 2016
REC opinion
Further Information Favourable Opinion