A Safety Study of Intravitreal PP-001 in Patients With Chronic, Non-Infectious Uveitis

Phase 1

Completed

• Conditions: Non-infectious Uveitis
• Interventions: Drug: PP-001

• Registration Number: NCT03634475
• Lead Sponsor: Panoptes Pharma GmbH

Brief Summary

In this study, PP-001 is assessed for safety and efficacy in patients diagnosed with non-infectious chronic uveitis. PP-001 is a novel small molecule that inhibits a specific enzyme (Dihydroorotate Dehydrogenase) and has shown pre-clinical efficacy in treatment of non-infectious uveitis. PP-001 will be administered to participants as a single intravitreal injection in ascending doses.

Detailed Description

This study assesses safety and efficacy of PP-001 after intravitreal injection in patients diagnosed with non-infectious chronic uveitis. PP-001 is a novel small molecule inhibitor of Dihydroorotate Dehydrogenase (DHODH) and has shown pre-clinical efficacy in treatment of non-infectious uveitis. PP-001 is to offer a local treatment alternative to corticosteroids with the objective to reduce or possibly replace steroid intake of non-infectious uveitis patients. In the current study, PP-001 will be injected in ascending doses into the vitreous body of the eye of study participants who suffer from non-infectious uveitis. Participants will be monitored for safety and efficacy evaluation of the study drug.

Study Timeline

• Study Start: 2017-01
• Study First Submitted: 2018-08-07
• Study First Submitted QC: 2018-08-14
• Study First Posted: 2018-08-16
• Primary Completion: 2018-11
• Study Completion: 2019-04
• Results First Submitted: 2023-12-21
• Status Verified: 2024-07
• Results First Submitted QC: 2024-07-15
• Last Update Submitted: 2024-07-15
• Results First Posted: 2024-10-09
• Last Update Posted: 2024-10-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

• Male or female patients at the age of 18 years or older who have diagnosis of chronic, posterior uveitis, intermediate uveitis or panuveitis

Exclusion Criteria

• Patients receiving specific medication/interventions as specified per protocol
• Pregnant or nursing patients

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
PP-001	PP-001	Single intravitreal injection of 3 up to 4 doses of PP-001

Primary Outcome Measures

Name	Time	Method
Evaluation of Safety and Tolerability by Determining Treatment Emergent Adverse Events	40 days	To assess safety and tolerability of ascending doses of PP-001 in participants with chronic, non-infectious uveitis when administered as single intravitreal injection

Trial Locations

Locations (9)

University Hospital Leuven; 🇧🇪 Leuven, Belgium

Charité Berlin; 🇩🇪 Berlin, Germany

LMU Munich; 🇩🇪 Munich, Germany

University Hospital Vienna; 🇦🇹 Vienna, Austria

Bristol Eye Hospital; 🇬🇧 Bristol, United Kingdom

Moorefields Eye Hospital; 🇬🇧 Moorfields, United Kingdom

University Hospital Muenster; 🇩🇪 Muenster, Germany

Rotterdam Eye Hospital; 🇳🇱 Rotterdam, Netherlands

University Hospital Tuebingen; 🇩🇪 Tuebingen, Germany