A Study in Subjects With Retinitis Pigmentosa

Study Overview
=================
Brief Summary
-----------------
An open-label Phase II clinical trial, 8 (eight) subjects with retinitis pigmentosa due to rhodopsin mutations (including P23H) will be identified and treated with serial intravitreal injections of ADX-2191 in the worse seeing eye. Ocular structure and function will be evaluated.

Official Title
-----------------
An Open-label, Phase II Study of ADX-2191 in Subjects With Retinitis Pigmentosa

Conditions
-----------------
Retinitis Pigmentosa

Intervention / Treatment
-----------------
* Drug: ADX-2191
* Drug: ADX-2191


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Up to 8 adult patients age 18 or older Diagnosis of retinitis pigmentosa due to rhodopsin gene mutation, including P23H Impairment on Visual Field as determined by perimetry Exclusion Criteria: Age < 18 years Pregnant Previous inflammatory/infectious events involving the eyes

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ADX-2191 Three Injections<br> | Drug: ADX-2191<br>* Monthly intravitreal injections of ADX-2191 (400 µg in 0.05 mL) for a total of three injections. Evaluation through week 16.<br>|
| Experimental: ADX-2191 Six Injections<br> | Drug: ADX-2191<br>* Monthly intravitreal injections of ADX-2191 (400 µg in 0.05 mL) for a total of six injections. Evaluation through week 16.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with adverse events (AEs) | Assessing the safety of ADX-2191 in patients with retinitis pigmentosa from AE collection | 16 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in visual acuity | Assessing the change in visual acuity in patients with retinitis pigmentosa treated with ADX-2191 using the Early Treatment Diabetic Retinopathy (ETDRS) chart. | 16 weeks | 
| Central retinal sensitivity | Assessing the central retinal sensitivity in patients using microperimetry, measured in decibels | 16 weeks | 
| Change in dark-adapted retinal sensitivity | Assessing the change in dark-adapted retinal sensitivity in patients using dark adapted chromatic perimetry, measured in decibels | 16 weeks | 
| Assessment for change in central subfield foveal thickness and ellipsoid zone area/width | Assessing the change in central subfield foveal thickness and ellipsoid zone area/width in patients using Spectral-Domain Optical Coherence Tomography (SD-OCT) as measured in square millimeters | 16 weeks | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
ADX-2191, Methotrexate, Retinitis Pigmentosa, Rhodopsin Mutation P23H