What side effects are associated with this type of intervention?

Gene therapy for Retinitis Pigmentosa, such as the AAV5-hRKp.RPGR vector, generally involves subretinal injections. Known side effects can include mild to moderate inflammation, retinal detachment, and potential infection at the injection site. Other common treatments for RP, like oral medications or vitamin supplements, may cause gastrointestinal discomfort or allergic reactions. It is crucial to discuss potential risks and benefits with a healthcare provider.

What prior FDA approvals exist?

The FDA has approved gene therapy for Retinitis Pigmentosa, specifically targeting the RPE65 gene. This gene therapy, known as voretigene neparvovec-rzyl (Luxturna), introduces a functional copy of the RPE65 gene to restore vision in patients with mutations in this gene. This approach is similar to the AAV5-hRKp.RPGR vector being studied, which aims to introduce a functional RPGR gene to treat X-linked Retinitis Pigmentosa. These therapies represent significant advancements in treating genetic causes of RP by addressing the underlying genetic defects.

What other types of research exist?

Promising alternatives to the AAV5-hRKp.RPGR vector for Retinitis Pigmentosa patients include: 1) CRISPR-Cas9 based gene editing, which offers precise correction of genetic mutations; 2) RNA-based therapies such as antisense oligonucleotides (ASOs) that can modulate gene expression; 3) Optogenetics, which involves introducing light-sensitive proteins to restore vision; and 4) Stem cell therapy, which aims to replace damaged retinal cells with healthy ones derived from stem cells. These approaches are in various stages of research and clinical trials, showing potential for future treatment options.

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Gene Therapy

Gene Therapy for Retinitis Pigmentosa

Phase 3

Waitlist Available

Led By James Bainbridge, MD

Research Sponsored by MeiraGTx UK II Ltd

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or female, 3 years of age or older, has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory.

Must not have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline- month 60

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial tests a gene therapy for people with X-linked retinitis pigmentosa. It uses a harmless virus to deliver a healthy gene to eye cells, aiming to improve their function and slow down vision loss. Gene therapy targeting the RPE65 gene has shown promise in treating inherited retinal dystrophies, including retinitis pigmentosa.

Who is the study for?

This trial is for males and females aged 3 or older with X-linked retinitis pigmentosa (XLRP) confirmed by a specialist. Participants must have a specific genetic change in the RPGR gene verified by an accredited lab.

What is being tested?

The study tests two doses of AAV5-RPGR, a gene therapy vector, to treat XLRP. Patients will receive one of the two doses to see how well it works and what side effects occur.

What are the potential side effects?

Potential side effects are not specified here but may include typical reactions related to gene therapy such as immune response, eye irritation or inflammation, and vision changes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am 3 or older with XLRP confirmed by a specialist and a genetic test.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

N/A

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline- month 60

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline- month 60

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline- month 60 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from baseline in vision-guided mobility assessment (VMA), as measured by the ability of the participant to navigate through a VMA maze, after bilateral subretinal delivery of AAV5-hRKp.RPGR

Number of Participants With Abnormalities in Laboratory Assessments

Number of participants with Ocular and Non-ocular Adverse Events of AAV5-hRKp.RPGR in Participants with RPGR-XLRP

Secondary study objectives

Change From Baseline in Best Corrected Visual Acuity (BCVA) by ETDRS Chart Letter Score in Monocular Assessment

Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Worse-seeing Eye

Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment

+9 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Deferred Treatment From MGT-RPGR-021 of Intermediate DoseExperimental Treatment1 Intervention

Deferred treatment

Group II: Deferred Treatment From MGT-RPGR-021 Low DoseExperimental Treatment1 Intervention

Deferred treatment

Group III: Already Treated in MGT-RPGR-021Experimental Treatment2 Interventions

Already treated

0 / 2Eligibility 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, such as the AAV5-hRKp.RPGR vector, works by introducing a functional copy of the defective RPGR gene into retinal cells. This approach aims to restore normal gene function, thereby halting or slowing the progression of the disease. This is particularly important for RP patients as it targets the underlying genetic cause, offering the potential for more effective and long-lasting treatment compared to traditional symptom management.