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

NCT ID: NCT03634475

Last Updated: 2024-10-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-01-31
• Study Completion Date: 2019-04-30

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.

Conditions

Non-infectious Uveitis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

PP-001

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

Group Type: EXPERIMENTAL

PP-001

Intervention Type: DRUG

Drug - no placebo

Interventions

PP-001

Drug - no placebo

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Panoptes Pharma GmbH

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Panoptes Study Director

Role: STUDY_DIRECTOR

Panoptes Pharma

Locations

University Hospital Vienna

Vienna, , Austria

University Hospital Leuven

Leuven, , Belgium

Charité Berlin

Berlin, , Germany

LMU Munich

Munich, , Germany

University Hospital Muenster

Münster, , Germany

University Hospital Tuebingen

Tübingen, , Germany

Rotterdam Eye Hospital

Rotterdam, , Netherlands

Bristol Eye Hospital

Bristol, , United Kingdom

Moorefields Eye Hospital

Moorfields, , United Kingdom

Countries

Austria; Belgium; Germany; Netherlands; United Kingdom

References

Thurau S, Deuter CME, Heiligenhaus A, Pleyer U, Van Calster J, Barisani-Asenbauer T, Obermayr F, Sperl S, Seda-Zehetner R, Wildner G. A new small molecule DHODH-inhibitor [KIO-100 (PP-001)] targeting activated T cells for intraocular treatment of uveitis - A phase I clinical trial. Front Med (Lausanne). 2022 Oct 17;9:1023224. doi: 10.3389/fmed.2022.1023224. eCollection 2022.

Reference Type: DERIVED

PMID: 36325389 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

PP-001-1001

Identifier Type: -

Identifier Source: org_study_id