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

Phase 1

Completed

• Conditions: Keratoconjunctivitis; Ocular Surface Disease
• Interventions: Drug: PP-001; Other: Placebo

• Registration Number: NCT03769454
• Lead Sponsor: Panoptes Pharma GmbH

Brief Summary

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).

The study was amended and now includes patients with ocular surface inflammation.

Detailed Description

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 (cohorts 1-3) and in patients with ocular surface inflammation (cohort 4). PP-001 is a novel small molecule inhibitor of Dihydroorotate Dehydrogenase (DHODH) with anti-inflammatory properties.

Study Timeline

• Study Start: 2018-11-20
• Study First Submitted: 2018-12-06
• Study First Submitted QC: 2018-12-06
• Study First Posted: 2018-12-07
• Primary Completion: 2021-09-15
• Study Completion: 2022-03-15
• Status Verified: 2024-01
• Last Update Submitted: 2024-01-09
• Last Update Posted: 2024-01-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 45

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
PP-001 verum - cohort 4	PP-001	PP-001 verum - cohort 4
Placebo - cohort 1	Placebo	Placebo - cohort 1
PP-001 verum - cohort 3	PP-001	PP-001 verum - cohort 3
Placebo - cohort 2	Placebo	Placebo - cohort 2
Placebo - cohort 3	Placebo	Placebo - cohort 3
PP-001 verum - cohort 1	PP-001	PP-001 verum - cohort 1
Placebo - cohort 4	Placebo	Placebo - cohort 4
PP-001 verum - cohort 2	PP-001	PP-001 verum - cohort 2

Primary Outcome Measures

Name	Time	Method
Evaluation of safety and tolerability by determining treatment emergent adverse events	28 days (cohorts 1-3) and 20 days (cohort 4)	To assess safety and tolerability of ascending, multiple daily doses of PP-001 eye drops in healthy, adult volunteers and in patients with ocular surface inflammation

Secondary Outcome Measures

Name	Time	Method
Evaluation of Peak Plasma Concentration (Cmax) in peripheral blood	1 and 12 days (cohorts 1-3)	To evaluate the pharmacokinetics (Cmax) of PP-001 in healthy, adult volunteers following single and multiple daily ocular instillation
Evaluation of area under the plasma concentration versus time curve (AUC) in peripheral blood	1 and 12 days (cohorts 1-3)	To evaluate the pharmacokinetics (AUC) of PP-001 in healthy, adult volunteers following single and multiple daily ocular instillation

Trial Locations

Locations (1)

University Hospital Vienna; 🇦🇹 Vienna, Austria