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

NCT03769454 | Completed Phase 1 DMC

• 1 other identifier: PP-001-1101
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 1

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.

Conditions

Keratoconjunctivitis; Ocular Surface Disease

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Evaluation of Peak Plasma Concentration (Cmax) in peripheral blood

  1 and 12 days (cohorts 1-3)

• Evaluation of area under the plasma concentration versus time curve (AUC) in peripheral blood

  1 and 12 days (cohorts 1-3)

Study Arms (8)

PP-001 verum - cohort 1

EXPERIMENTAL

PP-001 verum - cohort 1

Drug: PP-001

Placebo - cohort 1

PLACEBO COMPARATOR

Placebo - cohort 1

Other: Placebo

PP-001 verum - cohort 2

EXPERIMENTAL

PP-001 verum - cohort 2

Drug: PP-001

Placebo - cohort 2

PLACEBO COMPARATOR

Placebo - cohort 2

Other: Placebo

PP-001 verum - cohort 3

EXPERIMENTAL

PP-001 verum - cohort 3

Drug: PP-001

Placebo - cohort 3

PLACEBO COMPARATOR

Placebo - cohort 3

Other: Placebo

PP-001 verum - cohort 4

EXPERIMENTAL

PP-001 verum - cohort 4

Drug: PP-001

Placebo - cohort 4

PLACEBO COMPARATOR

Placebo - cohort 4

Other: Placebo

Interventions

PP-001 DRUG

PP-001 eye drops

PP-001 verum - cohort 1; PP-001 verum - cohort 2; PP-001 verum - cohort 3; PP-001 verum - cohort 4

Placebo OTHER

Placebo eye drops

Placebo - cohort 1; Placebo - cohort 2; Placebo - cohort 3; Placebo - cohort 4

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Panoptes Pharma GmbH: lead

Study Sites (1)

University Hospital Vienna

Vienna, Austria

Location

MeSH Terms

Conditions

Keratoconjunctivitis

Condition Hierarchy (Ancestors)

Conjunctivitis; Conjunctival Diseases; Eye Diseases; Keratitis; Corneal Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 6, 2018
• First Posted: December 7, 2018
• Study Start: November 20, 2018
• Primary Completion: September 15, 2021
• Study Completion: March 15, 2022
• Last Updated: January 12, 2024

Record last verified: 2024-01

Locations

AU (1)