Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
RESTORE
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]
1 other identifier
interventional
27
2 countries
6
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jul 2021
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 15, 2021
CompletedFirst Posted
Study publicly available on registry
June 30, 2021
CompletedStudy Start
First participant enrolled
July 13, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 27, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
January 18, 2024
CompletedMarch 22, 2024
March 1, 2024
1.6 years
June 15, 2021
March 20, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
Secondary Outcomes (6)
Efficacy of MCO-010 as assessed by best corrected visual acuity.
Week 76
Efficacy of MCO-010 as assessed by mobility testing.
Weeks 16,24,32,52,76,100
Efficacy of MCO-010 as assessed by mobility testing.
Weeks 16,24,32,52,76,100
Efficacy of MCO-010 as assessed by static shape recognition assay.
Weeks 16,24,32,52,76,100
Efficacy of MCO-010 as assessed by static shape recognition assay.
Weeks 16,24,32,52,76,100
- +1 more secondary outcomes
Other Outcomes (2)
Efficacy of MCO-010 as assessed by a composite of functional assessments.
Week 52
Safety of MCO-010.
100 weeks
Study Arms (3)
MCO-010- High Dose
EXPERIMENTALParticipants receive 1.2E11gc/eye of MCO-010
MCO-010- Low Dose
EXPERIMENTALParticipants receive 0.9E11gc/eye of MCO-010
Sham Injection
SHAM COMPARATORParticipants receive sham injection
Interventions
The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
Eligibility Criteria
You may qualify if:
- Age ≥ 18 years
- Able to comprehend and give informed consent.
- Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing.
- 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.
You may not qualify if:
- Subjects are excluded from the study if any of the following criteria apply:
- Prior participation in gene therapy program
- 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).
- Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function.
- Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
- Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (6)
Nanoscope Clinical Site
Beverly Hills, California, 90211, United States
Nanoscope Clinical Site
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
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Dr Samarendra Mohanty
Nanoscope Therapeutics Inc.
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
- Masking Details
- Treatment assignment will be unknown (or masked) to the study participants, the evaluating physician (non-injecting), outcomes assessor, the sponsor and its agents.
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 15, 2021
First Posted
June 30, 2021
Study Start
July 13, 2021
Primary Completion
February 27, 2023
Study Completion
January 18, 2024
Last Updated
March 22, 2024
Record last verified: 2024-03
Data Sharing
- IPD Sharing
- Will share
- Time Frame
- 12 months after the study is completed
- Access Criteria
- Efficacy and Safety Results
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.