PP-001-1001
Research type
Research Study
Full title
A safety study of intravitreal administration of PP-001 in patients with chronic non-infectious uveitis having chronic inflammation
IRAS ID
242365
Contact name
Adam Ross
Contact email
Sponsor organisation
Panoptes Pharma Ges.m.b.H.
Eudract number
2016-000412-15
Duration of Study in the UK
0 years, 4 months, 31 days
Research summary
In the present study, PP-001 is assessed for safety and tolerability in patients diagnosed with non-infectious chronic uveitis. PP-001 is a novel small molecule that inhibits a specific enzyme and has shown preclinical efficacy in treatment of non-infectious uveitis. Due to its mode of action, PP-001 specifically targets the underlying cause of the disease which is characterized by chronic intraocular inflammation. Non-infectious uveitis covers uveitis cases considered to be of autoimmune or autoinflammatory origin. Current treatment ranges from topical therapy (commonly corticosteroid eye drops) to systemic immunosuppression. Topical medication is generally only used to treat uveitis affecting the anterior part of the eye. Patients suffering from intermediate and/or posterior uveitis are routinely treated with high doses of systemic corticosteroids. PP-001 is to offer a local treatment alternative to corticosteroids with the objective to reduce or possibly replace steroid intake. In this study, PP-001 will be injected in the vitreous body of the eye of patients who suffer from non-infectious uveitis. The study is designed as prospective, multi-centre, open-label, non-randomized, consecutive study. Eighteen patients of 18 years or older will be enrolled. The study will involve seven patient visits and two telephone calls over a total duration of 40 days. Patients will be divided into three cohorts of four patients and will receive 0.3, 0.6, 1.2 or 2.1 µg of PP-001 as a single intravitreal injection in one eye. The patients within a cohort will be dosed consecutively with a minimum time interval of 7 days between injections and results from each dosing will be reviewed before progressing with the subsequent dosing. The next cohort will only commence after the previous cohort has completed all study sessions up to day 28 and no safety issues have been identified. The sponsor of this study is Panoptes Pharma GmbH located in Vienna, Austria.
REC name
North East - York Research Ethics Committee
REC reference
18/NE/0108
Date of REC Opinion
21 May 2018
REC opinion
Further Information Favourable Opinion