Eye Diseases, Eye Diseases, Hereditary, Retinal Degeneration, Retinal Diseases, Retinal Dystrophies, Retinitis, Retinitis Pigmentosa
Conditions
Keywords
Retinitis Pigmentosa, Eye Diseases Hereditary, Eye Diseases, Retinal Degeneration, Inherited Retinal Diseases, Rod & cone dystrophies, Optogenetics, Gene Therapy, AAV vectors, Intravitreal Injections, Low Vision, Multi-Characteristic Opsin, No Light Perception, Visual Acuity, Multi-Luminance Y Mobility Test (MLYMT), Multi-Luminance Shape Discrimination Test (MLSDT)
Brief summary
The purpose of the study is to evaluate the safety and efficacy of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (MCO-010).
Detailed description
This multicenter, randomized, double-masked, sham-controlled, dose-ranging study will evaluate 2 dose levels of MCO-010 in up to 18 subjects with retinitis pigmentosa (9 per dose). An additional nine subjects will receive sham injection. Subjects with a confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing will be considered for participation in this study. All subjects will continue to be assessed for 100 weeks following treatment with MCO-010.
Interventions
BIOLOGICALGene Therapy Product-MCO-010
The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
PROCEDURESham Injection
Sham Injection
Sponsors
Nanoscope Therapeutics Inc.
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Masking description
Treatment assignment will be unknown (or masked) to the study participants, the evaluating physician (non-injecting), outcomes assessor, the sponsor and its agents.
Intervention model description
Following a 1:1:1 block randomization schema, 9 subjects will be enrolled in each MCO-010 treatment group, and 9 subjects will be enrolled in the sham-controlled group.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Age ≥ 18 years 2. Able to comprehend and give informed consent. 3. Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing. 4. Best-Corrected (Freiburg) Visual Acuity worse than 1.9 LogMAR (Snellen equivalent 20/1600) in the study eye and no better than 1.6 LogMAR (Snellen equivalent 20/800) in the fellow eye during screening.
Exclusion criteria
Subjects are excluded from the study if any of the following criteria apply: 1. Prior participation in gene therapy program 2. Pre-existing conditions in the study eye such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, active uveitis, corneal or lenticular opacities). 3. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function. 4. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis. 5. Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity. | Week 52 | Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 52. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of MCO-010 as assessed by best corrected visual acuity. | Week 76 | Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 76. |
| Efficacy of MCO-010 as assessed by mobility testing. | Weeks 16,24,32,52,76,100 | Change from Baseline in Multi-Luminance Y-Mobility Test score. Range: -1 to 5, higher score means better outcome. |
| Efficacy of MCO-010 as assessed by static shape recognition assay. | Weeks 16,24,32,52,76,100 | Proportion of subjects with Multi-Luminance Shape Discrimination Test scores of 2 or more light level improvements from Baseline. Range: 0% to 100%, higher score means better outcome. |
| Efficacy of MCO-010 as assessed on visual field. | Weeks 16,24,32,52,76,100 | Change from Baseline in Visual Fields measured by Humphrey 30-2 perimetry. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of MCO-010 as assessed by a composite of functional assessments. | Week 52 | Proportion of subjects demonstrating a ≥2 unit improvement from Baseline in EITHER the MLYMT OR the MLSDT score at Week 52. |
| Safety of MCO-010. | 100 weeks | Incidences, nature, and severity of ocular and non-ocular treatment emergent adverse events (TEAEs). |
Countries
Puerto Rico, United States
Outcome results
None listed