A Safety Study of Intravitreal PP-001 in Patients With Chronic, Non-Infectious Uveitis

Panoptes Pharma

Status and phase: Completed; Phase 2; Phase 1

Conditions: Non-infectious Uveitis

Treatments: Drug: PP-001

Study type: Interventional

Funder types: Industry

Identifiers

NCT03634475

PP-001-1001

Details and patient eligibility

About

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.

Full 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.

Enrollment: 12 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

12 participants in 1 patient group

PP-001

Experimental group

Description:

Single intravitreal injection of 3 up to 4 doses of PP-001

Treatment:

Drug: PP-001