VAPAUS UK Amendment #1
Research type
Research Study
Full title
A multicenter, Phase 3, Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the saftey and efficacy of PTX-022 in the treatment of moderate to severe Pachyonychia Congenita
IRAS ID
302650
Contact name
Edel O' Toole
Contact email
Sponsor organisation
Palvella Therapeutics, Inc
Eudract number
2021-003136-10
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
, PALV-05
Duration of Study in the UK
0 years, 11 months, 2 days
Research summary
Pachyonychia congenita is a genetic disorder for which there is no approved treatment. Patients have calluses on their feet with severe pain, overgrowth of the fingers and toe nails, white thickening inside the mouth and on the tongue and increased sweating on the feet. This leads to negative psychosocial effects and patients report that the pain and its impact on daily activities associated with standing and walking is the most distressing side effect of the disorder. In
this study a novel formulation of topical Sirolimus (3.9%, Gel) shall be investigated. It is expected to adequately penetrate the skin and reach the mutated keratinocytes which cause the above described complaints. Furthermore, systemic side effects that appear when taking Sirolimus orally, are deemed to be minimised. It is expected to decrease patients's complaints and thereby to increase the patient's overall quality of life by using this new formulation.REC name
London - City & East Research Ethics Committee
REC reference
22/LO/0012
Date of REC Opinion
26 Jan 2022
REC opinion
Further Information Favourable Opinion