Clinical trial NCT03634475: A Safety Study of Intravitreal PP-001 in Patients With Chronic, Non-Infectious Uveitis
Official title: A Safety Study of Intravitreal PP-001 in Patients With Chronic, Non-Infectious Uveitis Having Chronic Inflammation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Non-infectious Uveitis
Lead sponsor: Panoptes Pharma GmbH
Sponsor class: INDUSTRY
First posted: 2018-08-16
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* Male or female patients at the age of 18 years or older who have diagnosis of chronic, posterior uveitis, intermediate uveitis or panuveitis

Exclusion Criteria:

* Patients receiving specific medication/interventions as specified per protocol
* Pregnant or nursing patients

Primary outcomes:
measure: Evaluation of Safety and Tolerability by Determining Treatment Emergent Adverse Events
timeFrame: 40 days
Secondary outcomes:
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: University Hospital Vienna
city: Leuven
country: Belgium
facility: University Hospital Leuven
city: Berlin
country: Germany
facility: Charité Berlin
city: Muenster
country: Germany
facility: University Hospital Muenster
city: Munich
country: Germany
facility: LMU Munich
city: Tuebingen
country: Germany
facility: University Hospital Tuebingen
city: Rotterdam
country: Netherlands
facility: Rotterdam Eye Hospital
city: Bristol
country: United Kingdom
facility: Bristol Eye Hospital
city: Moorfields
country: United Kingdom
facility: Moorefields Eye Hospital
Study officials:
affiliation: Panoptes Pharma
name: Panoptes Study Director
role: STUDY_DIRECTOR
References:
citation: 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.