Gene Therapy for Retinitis Pigmentosa

Phase 2

Recruiting

Research Sponsored by Janssen Research & Development, LLC

Eligibility Criteria Checklist

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

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline up to 5.5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial aims to test whether delivering a specific gene therapy directly under the retina is safe and well-tolerated in adults and children with a specific type of genetic eye disorder called X-linked retinitis

Who is the study for?

This trial is for adults and children who have already been treated with AAV5-hRKp.RPGR gene therapy in a previous study (MGT009) and are either currently enrolled or have completed another study (MGT010). Participants must understand the study's purpose, procedures, and agree to follow the protocol.

What is being tested?

The trial is testing the safety of delivering a gene therapy called AAV5-hRKp.RPGR directly under the retina. It follows patients previously treated for X-linked retinitis pigmentosa to see how they do over time without additional intervention.

What are the potential side effects?

While specific side effects are not listed, typical concerns with subretinal injections may include eye irritation, inflammation, possible vision changes or disturbances. Gene therapies can also trigger immune responses.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline up to 5.5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline up to 5.5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline up to 5.5 years for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Number of Participants With Adverse Events (AE)

Number of Participants With Clinically Significant Laboratory Abnormalities

Secondary study objectives

Change from Baseline in Best Corrected Visual Acuity (BCVA) by ETDRS Chart Letter Scores

Change from Baseline in Contrast Sensitivity (log CS) as Measured by Pelli-Robson Chart

Change from Baseline in Low Luminance Visual Acuity (LLVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score

+5 more

Awards & Highlights

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: Cohort 3Experimental Treatment1 Intervention

The participants who are not willing to undergo surgery or are not eligible for surgery will be assessed in this cohort. They will be assessed yearly until 5 years after their initial eye surgery in previous study MGT009.

Group II: Cohort 2Experimental Treatment1 Intervention

Participants will receive the treatment dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 in the second eye once the safety will be determined in Cohort 1.

Group III: Cohort 1Experimental Treatment1 Intervention

Participants will receive a dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 depending on the dosage administered in study MGT009 (NCT03252847) in the past. After receiving the treatment, participants will be assessed for safety.

Do I qualify?Apply below to find out