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

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

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.

Study Overview

• Status: Completed
• Conditions: Keratoconjunctivitis; Ocular Surface Disease
• Intervention / Treatment: Other: Placebo; Drug: PP-001

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 Type: Interventional
• Enrollment (Actual): 45
• Phase: Phase 1

Contacts and Locations

Study Locations

• Austria
  • Vienna, Austria
    • University Hospital Vienna

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 64 years (Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria (excerpt):

Cohorts 1-3:

• 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

Cohort 4:

• male or female subjects 18-64 years of age with ocular surface inflammation in both eyes
• ocular surface inflammation as defined per protocol
• good general state of health

Exclusion Criteria (excerpt):

Cohorts 1-4:

• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

8

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PP-001 verum - cohort 1	Drug: PP-001; PP-001 eye drops
Placebo Comparator: Placebo - cohort 1	Other: Placebo; Placebo eye drops
Experimental: PP-001 verum - cohort 2	Drug: PP-001; PP-001 eye drops
Placebo Comparator: Placebo - cohort 2	Other: Placebo; Placebo eye drops
Experimental: PP-001 verum - cohort 3	Drug: PP-001; PP-001 eye drops
Placebo Comparator: Placebo - cohort 3	Other: Placebo; Placebo eye drops
Experimental: PP-001 verum - cohort 4	Drug: PP-001; PP-001 eye drops
Placebo Comparator: Placebo - cohort 4	Other: Placebo; Placebo eye drops

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, multiple daily doses of PP-001 eye drops in healthy, adult volunteers and in patients with ocular surface inflammation	28 days (cohorts 1-3) and 20 days (cohort 4)

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 (cohorts 1-3)
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 (cohorts 1-3)

Collaborators and Investigators

• Sponsor: Panoptes Pharma GmbH

Study record dates

Study Major Dates

• Study Start (Actual): November 20, 2018
• Primary Completion (Actual): September 15, 2021
• Study Completion (Actual): March 15, 2022

Study Registration Dates

• First Submitted: December 6, 2018
• First Submitted That Met QC Criteria: December 6, 2018
• First Posted (Actual): December 7, 2018

Study Record Updates

• Last Update Posted (Actual): January 12, 2024
• Last Update Submitted That Met QC Criteria: January 9, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Conjunctivitis; Conjunctival Diseases; Keratitis; Corneal Diseases; Keratoconjunctivitis
• Other Study ID Numbers: PP-001-1101

Drug and device information, study documents

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