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

Study Overview
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Brief Summary
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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
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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.

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

Conditions
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Non-infectious Uveitis

Intervention / Treatment
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* Drug: PP-001


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PP-001<br>Single intravitreal injection of 3 up to 4 doses of PP-001 | Drug: PP-001<br>* Drug - no placebo<br>|

What is the study measuring?
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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 |