KeratoconjunctivitisPhase 1NCT03769454
A Phase I Study of PP-001 Eye Drops in Healthy Adult Volunteers
A Phase 1 study testing PP-001 in people with Keratoconjunctivitis and Ocular Surface Disease, run by Panoptes Pharma GmbH at 1 site in Austria.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 64 yearsHealthy volunteers can take part
- Conditions the study is for
- Keratoconjunctivitis, Ocular Surface Disease
- What is being tested?
- PP-001
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 45 participants
- Where
- 1 siteAustria
- Study dates
- 20 Nov 2018to 15 Mar 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 64 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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.
- Conditions
- Keratoconjunctivitis, Ocular Surface Disease
- Being tested
- PP-001, Placebo
- Medical subject headings
- Keratoconjunctivitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PP-001Drug
PP-001 eye drops
PlaceboOther
Placebo eye drops
| Study group | Role | What they receive |
|---|---|---|
| PP-001 verum - cohort 1 | Experimental | PP-001 verum - cohort 1 Receives: Drug: PP-001 |
| Placebo - cohort 1 | Placebo comparator | Placebo - cohort 1 Receives: Other: Placebo |
| PP-001 verum - cohort 2 | Experimental | PP-001 verum - cohort 2 Receives: Drug: PP-001 |
| Placebo - cohort 2 | Placebo comparator | Placebo - cohort 2 Receives: Other: Placebo |
| PP-001 verum - cohort 3 | Experimental | PP-001 verum - cohort 3 Receives: Drug: PP-001 |
| Placebo - cohort 3 | Placebo comparator | Placebo - cohort 3 Receives: Other: Placebo |
| PP-001 verum - cohort 4 | Experimental | PP-001 verum - cohort 4 Receives: Drug: PP-001 |
| Placebo - cohort 4 | Placebo comparator | Placebo - cohort 4 Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 28 days (cohorts 1-3) and 20 days (cohort 4)
Secondary outcomes (2)
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
Measured: 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
Measured: 1 and 12 days (cohorts 1-3)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2018
- Study start20 Nov 2018
- Primary completion15 Sep 2021
- Study completion15 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for KeratoconjunctivitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Panoptes Pharma GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03769454
- Sponsor's protocol ID
- PP-001-1101
- Status last verified
- Jan 2024
- First submitted
- 6 Dec 2018
- Last updated
- 12 Jan 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Safety and Tolerability Study of PP-001 Eye Drops in Healthy Adult Volunteers
- ClinicalTrials.gov ID
- NCT03769454
- Acronym / sponsor ID
- PP-001-1101
- Conditions
- Keratoconjunctivitis, Ocular Surface Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Panoptes Pharma GmbHIndustry (pharma, biotech, devices)
- Study start
- 20 Nov 2018 (actual)
- Primary completion
- 15 Sep 2021 (actual)
- Study completion
- 15 Mar 2022 (actual)
- First posted
- 7 Dec 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 8
| Arm | Type | Receives |
|---|---|---|
| PP-001 verum - cohort 1 | Experimental | Drug: PP-001PP-001 verum - cohort 1 |
| Placebo - cohort 1 | Placebo comparator | Other: PlaceboPlacebo - cohort 1 |
| PP-001 verum - cohort 2 | Experimental | Drug: PP-001PP-001 verum - cohort 2 |
| Placebo - cohort 2 | Placebo comparator | Other: PlaceboPlacebo - cohort 2 |
| PP-001 verum - cohort 3 | Experimental | Drug: PP-001PP-001 verum - cohort 3 |
| Placebo - cohort 3 | Placebo comparator | Other: PlaceboPlacebo - cohort 3 |
| PP-001 verum - cohort 4 | Experimental | Drug: PP-001PP-001 verum - cohort 4 |
| Placebo - cohort 4 | Placebo comparator | Other: PlaceboPlacebo - cohort 4 |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- 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
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PP-001PP-001 eye drops | Drug | PP-001 verum - cohort 1, PP-001 verum - cohort 2, PP-001 verum - cohort 3, PP-001 verum - cohort 4 |
| PlaceboPlacebo eye drops | Other | Placebo - cohort 1, Placebo - cohort 2, Placebo - cohort 3, Placebo - cohort 4 |
Primary outcome
Evaluation of safety and tolerability by determining treatment emergent adverse events
Time frame: 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
2 secondary outcomes
Evaluation of Peak Plasma Concentration (Cmax) in peripheral blood
Time frame: 1 and 12 days (cohorts 1-3)
Evaluation of area under the plasma concentration versus time curve (AUC) in peripheral blood
Time frame: 1 and 12 days (cohorts 1-3)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jan 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PP-001 in people with Keratoconjunctivitis and Ocular Surface Disease, run by Panoptes Pharma GmbH at 1 site in Austria.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Austria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of safety and tolerability by determining treatment emergent adverse events (time frame: 28 days (cohorts 1-3) and 20 days (cohort 4)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jan 2024.