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Gene Therapy

Gene Therapy for Retinitis Pigmentosa (RESTORE Trial)

Phase 2
Waitlist Available
Research Sponsored by Nanoscope Therapeutics Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age ≥ 18 years
Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing.
Must not have
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.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up weeks 16,24,32,52,76,100

Summary

This trial tests a new eye injection that uses a virus to deliver a helpful protein to improve vision in patients with severe vision loss from Advanced Retinitis Pigmentosa.

Who is the study for?
Adults over 18 with advanced Retinitis Pigmentosa (RP) and very poor vision can join this trial. They must have a confirmed diagnosis of RP, some remaining retinal layers visible on OCT scans, and understand the study to give consent. Excluded are those with other eye diseases like glaucoma, recent eye surgery, or infections like HIV; those who've had gene therapy before; or cannot perform mobility tests.
What is being tested?
The trial is testing MCO-010 optogenetic therapy involving one injection into the eye compared to a sham procedure. It aims to see if this gene therapy product can safely improve vision in people with severe visual impairment due to RP.
What are the potential side effects?
Potential side effects may include typical risks associated with intravitreal injections such as eye inflammation, increased intraocular pressure, cataracts formation, retinal detachment or bleeding inside the eye.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 18 years old or older.
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I have been diagnosed with advanced Retinitis Pigmentosa.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My study eye does not have glaucoma, significant vision loss, active inflammation, or cloudiness.
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I do not have any other serious illnesses that could affect my brain function.
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My eye condition prevents pupil dilation.
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I don't have eye conditions that could affect vision tests.
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I am not taking hydroxychloroquine, chloroquine, or similar drugs.
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I have ongoing eye inflammation or a history of eye inflammation without a known cause.
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I have received a retinal prosthesis or gene/stem cell therapy.
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I have not had eye surgery in the study eye within the last 3 months.
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My central vision is significantly affected due to retinal detachment in one eye.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~weeks 16,24,32,52,76,100
This trial's timeline: 3 weeks for screening, Varies for treatment, and weeks 16,24,32,52,76,100 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity.
Secondary study objectives
Efficacy of MCO-010 as assessed by best corrected visual acuity.
Efficacy of MCO-010 as assessed by mobility testing.
Efficacy of MCO-010 as assessed by static shape recognition assay.
+1 more
Other study objectives
Efficacy of MCO-010 as assessed by a composite of functional assessments.
Safety of MCO-010.

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: MCO-010- Low DoseExperimental Treatment1 Intervention
Participants receive 0.9E11gc/eye of MCO-010
Group II: MCO-010- High DoseExperimental Treatment1 Intervention
Participants receive 1.2E11gc/eye of MCO-010
Group III: Sham InjectionPlacebo Group1 Intervention
Participants receive sham injection
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Gene Therapy Product-MCO-010
2021
Completed Phase 2
~30

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Gene therapy for Retinitis Pigmentosa (RP) often involves using viral vectors to deliver functional genes or light-sensitive proteins to the retina. For example, the MCO-010 trial uses a viral vector to introduce a Multi-Characteristic Opsin, a light-sensitive protein, into retinal cells, aiming to restore vision by enabling these cells to respond to light. This approach is significant for RP patients as it targets the underlying genetic causes of the disease, potentially halting or reversing vision loss. Other common treatments include the use of carbonic anhydrase inhibitors to reduce macular edema and retinal prostheses to enhance visual function. These treatments are crucial as they offer hope for improved quality of life and functional vision in patients with RP.
Gene Therapy for the Retinal Degeneration of Usher Syndrome Caused by Mutations in MYO7A.Gene therapy for Leber congenital amaurosis: advances and future directions.Restoration of vision in the pde6β-deficient dog, a large animal model of rod-cone dystrophy.

Find a Location

Who is running the clinical trial?

Nanoscope Therapeutics Inc.Lead Sponsor
6 Previous Clinical Trials
87 Total Patients Enrolled
4 Trials studying Retinitis Pigmentosa
75 Patients Enrolled for Retinitis Pigmentosa
Aaron Osborne, MDStudy ChairNanoscope Therapeutics Inc.
1 Previous Clinical Trials
6 Total Patients Enrolled
Mohamed Genead, MDStudy DirectorNanoscope Therapeutics Inc.

Media Library

MCO-010 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04945772 — Phase 2
Retinitis Pigmentosa Research Study Groups: MCO-010- Low Dose, MCO-010- High Dose, Sham Injection
Retinitis Pigmentosa Clinical Trial 2023: MCO-010 Highlights & Side Effects. Trial Name: NCT04945772 — Phase 2
MCO-010 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04945772 — Phase 2
Retinitis Pigmentosa Patient Testimony for trial: Trial Name: NCT04945772 — Phase 2
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