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

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

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.

Study Overview

• Status: Unknown
• Conditions: Non-infectious Uveitis
• Intervention / Treatment: Drug: PP-001

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 Type: Interventional
• Enrollment (Anticipated): 18
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Austria
  • Vienna, Austria
    • University Hospital Vienna
• Belgium
  • Leuven, Belgium
    • University Hospital Leuven
• Germany
  • Berlin, Germany
    • Charite Berlin
  • Muenster, Germany
    • University Hospital Muenster
  • Munich, Germany
    • LMU Munich
  • Tuebingen, Germany
    • University Hospital Tuebingen
• Netherlands
  • Rotterdam, Netherlands
    • Rotterdam Eye Hospital
• United Kingdom
  • Bristol, United Kingdom
    • Bristol Eye Hospital
  • Moorfields, United Kingdom
    • Moorefields Eye Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PP-001; Single intravitreal injection of 3 up to 4 doses of PP-001	Drug: PP-001; Drug - no placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of safety and tolerability by determining treatment emergent adverse events	To assess safety and tolerability of ascending doses of PP-001 in participants with chronic, non-infectious uveitis when administered as single intravitreal injection	40 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of improvement of ocular inflammation between baseline and follow-up visits using slit-lamp examinations	To assess the improvement of inflammation using slit lamp examinations in participants with chronic, non-infectious uveitis after ascending doses of PP-001 when administered as single intravitreal injection	40 days
Evaluation of improvement of ocular inflammation between baseline and follow-up visits using Optical Coherence Tomography (OCT) examinations	To assess the improvement of inflammation using OCT examinations in participants with chronic, non-infectious uveitis after ascending doses of PP-001 when administered as single intravitreal injection	40 days
Evaluation of Peak Plasma Concentration (Cmax) in peripheral blood	To evaluate the pharmacokinetics (Cmax) in participants with chronic, non-infectious uveitis after ascending doses of PP-001 when administered as single intravitreal injection	2 days
Evaluation of Area under the plasma concentration versus time curve (AUC) in peripheral blood	To evaluate the pharmacokinetics (AUC) in participants with chronic, non-infectious uveitis after ascending doses of PP-001 when administered as single intravitreal injection	2 days

Collaborators and Investigators

• Sponsor: Panoptes Pharma GmbH
• Investigators: Study Director: Panoptes Study Director, Panoptes Pharma

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2017
• Primary Completion (Anticipated): November 1, 2020
• Study Completion (Anticipated): December 1, 2020

Study Registration Dates

• First Submitted: August 7, 2018
• First Submitted That Met QC Criteria: August 14, 2018
• First Posted (Actual): August 16, 2018

Study Record Updates

• Last Update Posted (Actual): March 11, 2020
• Last Update Submitted That Met QC Criteria: March 10, 2020
• Last Verified: March 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Uveal Diseases; Uveitis
• Other Study ID Numbers: PP-001-1001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No