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

Non-infectious Uveitis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03634475
• Other study ID #: PP-001-1001
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: January 2017
• Est. completion date: December 2020

Study information

• Verified date: March 2020
• Source: Panoptes Pharma GmbH
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 18
• Est. completion date: December 2020
• Est. primary completion date: November 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Non-infectious Uveitis
• Uveitis

Intervention

Drug:
• PP-001
Drug - no placebo

Locations

Country	Name	City	State
Austria	University Hospital Vienna	Vienna
Belgium	University Hospital Leuven	Leuven
Germany	Charité Berlin	Berlin
Germany	University Hospital Muenster	Muenster
Germany	LMU Munich	Munich
Germany	University Hospital Tuebingen	Tuebingen
Netherlands	Rotterdam Eye Hospital	Rotterdam
United Kingdom	Bristol Eye Hospital	Bristol
United Kingdom	Moorefields Eye Hospital	Moorfields

Sponsors (1)

Lead Sponsor	Collaborator
Panoptes Pharma GmbH

Countries where clinical trial is conducted

Austria,  Belgium,  Germany,  Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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