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In this study, PP-001 is assessed for safety and efficacy in patients diagnosed with non-infectious chronic uveitis. PP-001 is a novel small molecule that inhibits a specific enzyme (Dihydroorotate Dehydrogenase) and has shown pre-clinical efficacy in treatment of non-infectious uveitis. PP-001 will be administered to participants as a single intravitreal injection in ascending doses.
This study assesses safety and efficacy of PP-001 after intravitreal injection in patients diagnosed with non-infectious chronic uveitis. PP-001 is a novel small molecule inhibitor of Dihydroorotate Dehydrogenase (DHODH) and has shown pre-clinical efficacy in treatment of non-infectious uveitis. PP-001 is to offer a local treatment alternative to corticosteroids with the objective to reduce or possibly replace steroid intake of non-infectious uveitis patients. In the current study, PP-001 will be injected in ascending doses into the vitreous body of the eye of study participants who suffer from non-infectious uveitis. Participants will be monitored for safety and efficacy evaluation of the study drug.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PP-001 | Experimental | Single intravitreal injection of 3 up to 4 doses of PP-001 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PP-001 | Drug | Drug - no placebo |
|
| Measure | Description | Time Frame |
|---|---|---|
| Evaluation of Safety and Tolerability by Determining Treatment Emergent Adverse Events | To assess safety and tolerability of ascending doses of PP-001 in participants with chronic, non-infectious uveitis when administered as single intravitreal injection | 40 days |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Panoptes Study Director | Panoptes Pharma | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Hospital Vienna | Vienna | Austria | ||||
| University Hospital Leuven |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36325389 | Derived | Thurau S, Deuter CME, Heiligenhaus A, Pleyer U, Van Calster J, Barisani-Asenbauer T, Obermayr F, Sperl S, Seda-Zehetner R, Wildner G. A new small molecule DHODH-inhibitor [KIO-100 (PP-001)] targeting activated T cells for intraocular treatment of uveitis - A phase I clinical trial. Front Med (Lausanne). 2022 Oct 17;9:1023224. doi: 10.3389/fmed.2022.1023224. eCollection 2022. |
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| ID | Title | Description |
|---|---|---|
| FG000 | PP-001 | Single intravitreal injection of 3 up to 4 doses of PP-001 PP-001: Drug - no placebo |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | PP-001 | Single intravitreal injection of 3 up to 4 doses of PP-001 PP-001: Drug - no placebo |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Evaluation of Safety and Tolerability by Determining Treatment Emergent Adverse Events | To assess safety and tolerability of ascending doses of PP-001 in participants with chronic, non-infectious uveitis when administered as single intravitreal injection | Posted | Count of Participants | Participants | 40 days |
|
|
14 days prior to dosing and 40 days after dosing
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | PP-001 | Single intravitreal injection of 3 up to 4 doses of PP-001 PP-001: Drug - no placebo |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Conjunctival haemorrhage | Eye disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Panoptes Pharma | +43 664 851 5865 | ssperl@kiorapharma.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 3, 2017 | Dec 14, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 17, 2020 | Dec 14, 2023 | SAP_001.pdf |
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| Leuven |
| Belgium |
| Charité Berlin | Berlin | Germany |
| LMU Munich | Munich | Germany |
| University Hospital Muenster | Münster | Germany |
| University Hospital Tuebingen | Tübingen | Germany |
| Rotterdam Eye Hospital | Rotterdam | Netherlands |
| Bristol Eye Hospital | Bristol | United Kingdom |
| Moorefields Eye Hospital | Moorfields | United Kingdom |
| Participants |
|
| Age, Continuous | Mean | Full Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
| 0 |
| 12 |
| 0 |
| 12 |
| 12 |
| 12 |
| Anterior Chamber Cell | Eye disorders | Systematic Assessment |
|
| Conjunctival hyperaemia | Eye disorders | Systematic Assessment |
|
| Anterior chamber flare | Eye disorders | Systematic Assessment |
|
| Eye irritation | Eye disorders | Systematic Assessment |
|
| Ocular hyperaemia | Eye disorders | Systematic Assessment |
|
| Punctate keratitis | Eye disorders | Systematic Assessment |
|
| Conjunctival cyst | Eye disorders | Systematic Assessment |
|
| Conjunctival oedema | Eye disorders | Systematic Assessment |
|
| Corneal deposits | Eye disorders | Systematic Assessment |
|
| Cystoid macular oedema | Eye disorders | Systematic Assessment |
|
| Episcleritis | Eye disorders | Systematic Assessment |
|
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