A Phase I Study of PP-001 Eye Drops in Healthy Adult Volunteers

Study Overview
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Brief Summary
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In this study, PP-001 eye drops are assessed for safety and tolerability in healthy, adult volunteers. PP-001 is a novel, anti-inflammatory small molecule that inhibits a specific enzyme (Dihydroorotate Dehydrogenase).

Detailed Description
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In this prospective, single-centre, double blind (within each cohort), placebo controlled, randomised study, safety and tolerability of PP-001 eye drops is assessed in healthy, adult volunteers. PP-001 is a novel small molecule inhibitor of Dihydroorotate Dehydrogenase (DHODH) with anti-inflammatory properties. PP-001 eye drops will be administered up to 4 times per day over a period of 13 days in ascending doses. Participants will be monitored for safety and tolerability evaluation of the study drug.

Official Title
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A Phase I Safety and Tolerability Study of PP-001 Eye Drops in Healthy Adult Volunteers

Conditions
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Keratoconjunctivitis

Intervention / Treatment
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* Drug: PP-001
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: male or female healthy volunteers 18 - 64 years of age good general state of health and participants must not have a diagnosis of any eye disease that could effect the pharmacokinetics of PP-001 Exclusion Criteria: participation in other clinical trials within 30 days prior to dosing start (ocular and non-ocular clinical trials) pregnant or nursing patients regular use of any ocular agents within 60 days prior to start dosing

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 64 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PP-001 low dose group<br> | Drug: PP-001<br>* PP-001 eye drops<br>|
| Placebo Comparator: Placebo low dose group<br> | Other: Placebo<br>* Placebo eye drops<br>|
| Experimental: PP-001 mid dose group<br> | Drug: PP-001<br>* PP-001 eye drops<br>|
| Placebo Comparator: Placebo mid dose group<br> | Other: Placebo<br>* Placebo eye drops<br>|
| Experimental: PP-001 high dose group<br> | Drug: PP-001<br>* PP-001 eye drops<br>|
| Placebo Comparator: Placebo high dose group<br> | Other: Placebo<br>* Placebo eye drops<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, multiple daily doses of PP-001 eye drops in healthy, adult volunteers | 28 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluation of Peak Plasma Concentration (Cmax) in peripheral blood | To evaluate the pharmacokinetics (Cmax) of PP-001 in healthy, adult volunteers following single and multiple daily ocular instillation | 1 and 12 days | 
| Evaluation of area under the plasma concentration versus time curve (AUC) in peripheral blood | To evaluate the pharmacokinetics (AUC) of PP-001 in healthy, adult volunteers following single and multiple daily ocular instillation | 1 and 12 days |