Retinitis Pigmentosa
Conditions
Keywords
ADX-2191, Methotrexate, Retinitis Pigmentosa, Rhodopsin Mutation P23H
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.
Interventions
DRUGADX-2191
Monthly intravitreal injections of ADX-2191 (400 µg in 0.05 mL) for a total of three injections. Evaluation through week 16.
Sponsors
Aldeyra Therapeutics, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Up to 8 adult patients age 18 or older 2. Diagnosis of retinitis pigmentosa due to rhodopsin gene mutation, including P23H 3. Impairment on Visual Field as determined by perimetry
Exclusion criteria
1. Age \< 18 years 2. Pregnant 3. Previous inflammatory/infectious events involving the eyes
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Subjects With Serious Adverse Events | The safety assessment period was 16 weeks. | The proportion of subjects that experience at least one serious adverse event |
Countries
United States
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 5 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 4 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants |
| 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 | 8 Participants |
| Region of Enrollment United States | 8 Participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 4 | 0 / 4 |
| other Total, other adverse events | 3 / 4 | 3 / 4 |
| serious Total, serious adverse events | 0 / 4 | 0 / 4 |
Outcome results
None listed