Clinical trial NCT04945772: Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
Official title: A Phase 2b Randomized, Double-Masked, Sham-Controlled, Study to Evaluate the Efficacy and Safety of Intravitreal Injection of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Retinitis Pigmentosa
Retinitis
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
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)
Lead sponsor: Nanoscope Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2021-06-30
Enrollment: 27
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
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.
Primary purpose: TREATMENT

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.

Eligibility criteria: 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).

Primary outcomes:
measure: Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity.
timeFrame: Week 52
Secondary outcomes:
measure: Efficacy of MCO-010 as assessed by best corrected visual acuity.
timeFrame: Week 76
measure: Efficacy of MCO-010 as assessed by mobility testing.
timeFrame: Weeks 16,24,32,52,76,100
measure: Efficacy of MCO-010 as assessed by mobility testing.
timeFrame: Weeks 16,24,32,52,76,100
measure: Efficacy of MCO-010 as assessed by static shape recognition assay.
timeFrame: Weeks 16,24,32,52,76,100
measure: Efficacy of MCO-010 as assessed by static shape recognition assay.
timeFrame: Weeks 16,24,32,52,76,100
measure: Efficacy of MCO-010 as assessed on visual field.
timeFrame: Weeks 16,24,32,52,76,100
Other outcomes:
measure: Efficacy of MCO-010 as assessed by a composite of functional assessments.
timeFrame: Week 52
measure: Safety of MCO-010.
timeFrame: 100 weeks
Locations:
city: Beverly Hills
country: United States
facility: Nanoscope Clinical Site
state: California
city: Pensacola
country: United States
facility: Nanoscope Clinical Site
state: Florida
city: Fargo
country: United States
facility: Nanoscope Clinical Site
state: North Dakota
city: Houston
country: United States
facility: Nanoscope Clinical Site
state: Texas
city: McAllen
country: United States
facility: Nanoscope Clinical Site
state: Texas
city: Arecibo
country: Puerto Rico
facility: Nanoscope Clinical Site
Study officials:
affiliation: Nanoscope Therapeutics Inc.
name: Dr Samarendra Mohanty
role: STUDY_CHAIR
References: