What side effects are associated with this type of intervention?

Common treatments for Retinitis Pigmentosa, particularly gene therapies using viral vectors like MCO-010, can have side effects primarily related to the surgical delivery of the treatment, such as inflammation or infection at the injection site. Immune responses to the viral vector can also cause inflammation or other immune-mediated reactions. Other treatments, such as vitamin A supplementation, are generally safe but do not significantly improve visual function.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Retinitis Pigmentosa that use gene therapy with viral vectors to deliver light-sensitive proteins to the eye. However, similar approaches have been explored in clinical trials, such as the use of self-complementary AAV-mediated gene therapy to restore visual function in models of Rpe65 deficiency, which is related to Leber's congenital amaurosis-2. These trials have shown promise in restoring visual function and preventing cone degeneration, emphasizing the potential of early intervention.

What other types of research exist?

Promising alternatives to MCO-010 for Retinitis Pigmentosa include: 1) Gene therapy targeting RPE65 mutations, which has shown consistent visual function improvements in clinical trials. 2) Cell-based therapies, such as the use of cardiosphere-derived cells and exosomes, which have demonstrated disease-modifying bioactivity in muscular dystrophy models and may have potential applications in retinal diseases. 3) Pharmacological treatments like RNA interference (RNAi) therapies and antisense oligonucleotides, which are being explored for their ability to modify disease progression at the genetic level.

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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 weeks3 visits

Treatment ~ Varies

Follow Up ~ weeks 16,24,32,52,76,100

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

0 / 11Eligibility criteria met

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.