Non-infectious Uveitis
Conditions
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.
Interventions
DRUGPP-001
Drug - no placebo
Sponsors
Panoptes Pharma GmbH
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evaluation of Safety and Tolerability by Determining Treatment Emergent Adverse Events | 40 days | To assess safety and tolerability of ascending doses of PP-001 in participants with chronic, non-infectious uveitis when administered as single intravitreal injection |
Countries
Austria, Belgium, Germany, Netherlands, United Kingdom
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| PP-001 Single intravitreal injection of 3 up to 4 doses of PP-001
PP-001: Drug - no placebo | 12 |
| Total | 12 |
Baseline characteristics
| Characteristic | PP-001 |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 11 Participants |
| Age, Continuous | 52 years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 12 Participants |
| Region of Enrollment Austria | 12 participants |
| Sex: Female, Male Female | 8 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 12 |
| other Total, other adverse events | 12 / 12 |
| serious Total, serious adverse events | 0 / 12 |
Outcome results
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
Time frame: 40 days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| PP-001 | Evaluation of Safety and Tolerability by Determining Treatment Emergent Adverse Events | 12 Participants |