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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MCO-010- High Dose | Experimental | Participants receive 1.2E11gc/eye of MCO-010 |
|
| MCO-010- Low Dose | Experimental | Participants receive 0.9E11gc/eye of MCO-010 |
|
| Sham Injection | Sham Comparator | Participants receive sham injection |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Gene Therapy Product-MCO-010 | Biological | The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette |
|
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity. | Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 52. | Week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy of MCO-010 as assessed by best corrected visual acuity. | Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 76. | Week 76 |
| Efficacy of MCO-010 as assessed by mobility testing. |
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy of MCO-010 as assessed by a composite of functional assessments. | Proportion of subjects demonstrating a ≥2 unit improvement from Baseline in EITHER the MLYMT OR the MLSDT score at Week 52. | Week 52 |
| Safety of MCO-010. |
Inclusion Criteria:
Exclusion Criteria:
Subjects are excluded from the study if any of the following criteria apply:
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| Name | Affiliation | Role |
|---|---|---|
| Dr Samarendra Mohanty | Nanoscope Therapeutics Inc. | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nanoscope Clinical Site | Beverly Hills | California | 90211 | United States | ||
| Nanoscope Clinical Site |
The results of the clinical trial will be made available when the study is completed. The results will be published on this site and be available to conference presentations and publications.
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12 months after the study is completed
Efficacy and Safety Results
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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.
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Treatment assignment will be unknown (or masked) to the study participants, the evaluating physician (non-injecting), outcomes assessor, the sponsor and its agents.
| Sham Injection | Procedure | Sham Injection |
|
Change from Baseline in Multi-Luminance Y-Mobility Test score. Range: -1 to 5, higher score means better outcome.
| Weeks 16,24,32,52,76,100 |
| Efficacy of MCO-010 as assessed by mobility testing. | Proportion of subjects with Multi-Luminance Y-Mobility Test score scores of 2 or more light level improvement from Baseline. Range: 0% to100%, higher score means better outcome. | Weeks 16,24,32,52,76,100 |
| Efficacy of MCO-010 as assessed by static shape recognition assay. | 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. | Weeks 16,24,32,52,76,100 |
| Efficacy of MCO-010 as assessed by static shape recognition assay. | Change from Baseline in Multi-Luminance Shape Discrimination Test score. Range: 0 to 5, higher score means better outcome. | Weeks 16,24,32,52,76,100 |
| Efficacy of MCO-010 as assessed on visual field. | Change from Baseline in Visual Fields measured by Humphrey 30-2 perimetry. | Weeks 16,24,32,52,76,100 |
Incidences, nature, and severity of ocular and non-ocular treatment emergent adverse events (TEAEs).
| 100 weeks |
| Pensacola |
| Florida |
| 32503 |
| United States |
| Nanoscope Clinical Site | Fargo | North Dakota | 58103 | United States |
| Nanoscope Clinical Site | Houston | Texas | 77030 | United States |
| Nanoscope Clinical Site | McAllen | Texas | 78503 | United States |
| Nanoscope Clinical Site | Arecibo | 00612 | Puerto Rico |
| ID | Term |
|---|---|
| D012174 | Retinitis Pigmentosa |
| D012173 | Retinitis |
| D012164 | Retinal Diseases |
| D005128 | Eye Diseases |
| D015785 | Eye Diseases, Hereditary |
| D058499 | Retinal Dystrophies |
| D012162 | Retinal Degeneration |
| D000071700 | Cone-Rod Dystrophies |
| D015354 | Vision, Low |
| ID | Term |
|---|---|
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D014786 | Vision Disorders |
| D012678 | Sensation Disorders |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C005703 | salicylhydroxamic acid |
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