Active not recruitingPHASE2INTERVENTIONAL

A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)

This study is a follow-up for people who have previously received a type of gene therapy for an inherited eye condition called X-linked Retinitis Pigmentosa (XLRP). This condition causes gradual loss of vision, mainly affecting boys and men. The main aim is to check the safety and how well people tolerate this gene therapy, called AAV5-hRKp.RPGR, when it's given to their second eye. Researchers want to see if the treatment continues to be safe and if it helps preserve vision in the other eye. It includes both children and adults who have been part of earlier studies with this gene therapy. There are no new exclusion criteria for this particular study, meaning it's mainly for those already familiar with the treatment.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Janssen Research & Development, LLC

Enrolment target

Start

10 Oct 2024

Estimated completion

24 Oct 2030

X-Linked Retinitis Pigmentosa

What is this study about?

This study is designed for individuals who have a specific inherited eye condition called X-linked Retinitis Pigmentosa, often shortened to XLRP. This condition mainly affects boys and men and gradually causes their vision to get worse over time. It's inherited, meaning it's passed down through families. Researchers are looking into a new type of treatment called gene therapy, which aims to correct the genetic problem that causes XLRP. This particular gene therapy is called AAV5-hRKp.RPGR.

This study isn't for people who are new to this treatment. Instead, it's a 'follow-on' study. This means it's for people who have already received this gene therapy in one eye in previous research studies. The main goal now is to see how safe this treatment is and how well people cope with it when it's given to their *second* eye. This helps doctors understand more about the long-term effects and if it can help improve or save vision in both eyes.

By carefully observing participants who receive the treatment in their second eye, and by continuing to monitor them over time, doctors can gather important information. This information is crucial for understanding the full potential of this gene therapy and deciding if it could become a widely available treatment for people with XLRP in the future.

Key takeaways

This is a follow-up study for gene therapy in XLRP patients.
It's for people who have already had the treatment in one eye.
The study assesses safety and tolerability of treating the *second* eye.
It includes both children and adult males with XLRP.
Participation involves eye treatment and long-term check-ups.
You can stop participating at any time.

Who may be eligible?

This study is specifically for male patients, both children and adults, who have already taken part in previous studies involving this gene therapy for X-linked Retinitis Pigmentosa (XLRP). You must have received the AAV5-hRKp.RPGR gene therapy in one eye already. If you haven't been part of those earlier studies, this particular follow-on study wouldn't be suitable for you.

To join, you need to sign a consent form. This form explains everything about the study clearly, so you understand what's involved and agree to take part. It's important that you also agree to follow the study's instructions and attend all the long-term check-ups.

There are no specific reasons that would automatically prevent you from joining this study, provided you meet the main criteria of having been in the previous related studies and agree to the terms.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you male?
Have you been diagnosed with X-linked Retinitis Pigmentosa (XLRP)?
Have you previously received the AAV5-hRKp.RPGR gene therapy in one eye in an earlier study (MGT009 or MGT010)?
Are you willing to sign a consent form and follow the study's rules?
Are you prepared for long-term follow-up appointments?

Answer every question to see your result.

What does participation involve?

Taking part in this study means you will receive the AAV5-hRKp.RPGR gene therapy in your second eye, following the treatment you've already had in your first eye. This involves a surgical procedure to deliver the gene therapy directly into your eye. After the procedure, you'll have regular follow-up appointments with the study doctors and nurses. These visits will involve various eye tests and check-ups to monitor your vision, the health of your eye, and to ensure the treatment is safe and well-tolerated. The total duration of your participation, including these follow-up assessments, will depend on what was agreed in the previous studies you were part of, as this is a continuation of that long-term monitoring. You won't be given any additional medication specifically for this study beyond the gene therapy itself and any standard care needed around the procedure.

Potential risks and benefits

The potential benefits of taking part include the opportunity to receive gene therapy in your second eye, which may help to slow down or prevent further vision loss from X-linked Retinitis Pigmentosa in that eye. While gene therapies hold promise, there are potential risks, as with any medical procedure. These could include risks associated with eye surgery or possible reactions to the gene therapy itself. These risks will be fully explained by the study team. Remember, you can choose to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (3)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Massachusetts Eye and Ear Infirmary
Verified postcode
Boston, United States
University of Michigan Kellogg Eye Center
Verified postcode
Ann Arbor, United States
Moorfields Eye Hospital
Verified postcode
London, United Kingdom

Common questions

What is X-linked Retinitis Pigmentosa (XLRP)?

XLRP is an inherited eye condition, mainly affecting males, that causes a gradual loss of vision over time.

Who can join this study?

This study is for males, both children and adults, who have already received this gene therapy in one eye through a previous study.

What is gene therapy?

Gene therapy is a new type of treatment that aims to fix the genetic problem causing a disease, in this case, XLRP.

Will I get the gene therapy in my second eye?

Yes, if you meet the criteria and choose to participate, you will receive the gene therapy in your other eye.

Can I leave the study at any time?

Yes, you are free to withdraw from the study at any point, and your medical care will not be affected.