Learn About Clinical Studies

• X-Linked Retinitis Pigmentosa (XLRP) is a severe form of Retinitis Pigmentosa (RP), a rare, inherited retinal degenerative disorder that causes blindness and accounts for around 10% of RP cases.
• XLRP can be caused by a mutation in a gene called Retinitis Pigmentosa GTPase Regulator (RPGR). A mutation in RPGR can result in loss of vision due to degeneration (breakdown) of special cells in the retina called photoreceptors, which are needed for vision.
• Early symptoms include loss of night vision in adolescence with progressive loss of peripheral vision (resulting in tunnel vision). Ultimately this leads to loss of central vision, with most patients becoming legally blind by the age of 45.

• A clinical trial is a type of research that doctors and scientists use to investigate whether a medical/drug therapy is safe and effective in humans.
• Participation in clinical trials is voluntary, and participants can stop at any time.

The study drug (AGTC-501) is designed to provide healthy copies of the mutated RPGR gene to cells in the retina (the thin layer of tissue that lines the inner surface of the back of the eye) to make RPGR protein.

The Vista clinical trial is for males between the ages of 12 and 50 who have X-linked retinitis pigmentosa (XLRP) caused by a mutation (or defect) in the RPGR gene. Individuals must meet certain criteria to qualify for the study. You may be asked to provide documentation or permission to review your medical records.

You or your child may be eligible for the Vista Clinical Trial if:

• Diagnosed with XLRP and have RPGR gene mutation (confirmed by genetic testing)
• Best Corrected Visual Acuity in at least one eye is between 20/32 and 20/200
• Male
• Between 12 and 50 years of age

• If eligibility criteria are met, participants will then attend 3 screening visits at a study site.
• Qualified participants will be randomly assigned to 1 of 2 treatment groups or the control group.
• Eligible participants in the treatment groups will undergo a procedure to receive an injection of AGTC-501 in one eye. The study doctor and study participants will not know which dose is given.
• Participants in the control group will not receive AGTC-501 initially, but they will have the option to receive AGTC-501 after 1 year if they meet eligibility requirements.

• Trial participation lasts from 2 to 6 years.
• Participation for the treatment groups lasts for 5 years.
• Participants in the control group will participate for at least 1 year before being eligible for treatment. If you are eligible and choose to receive the study drug, you may participate in the study for up to an additional 5 years.
• Participants who are ineligible or choose not to receive the study drug will participate for two years.

Control groups are very important in clinical research.

• For this study it will allow researchers to compare results from a group of participants who received the study drug to results from those who did not.
• This comparison will help researchers show whether the study drug had any effect.
• After at least one year, participants in the control group may choose to end participation or receive the study drug, if eligible.

• If you have been diagnosed with XLRP and have had the RPGR gene mutation confirmed through genetic testing, you may be eligible for the Vista clinical trial.
• If you are unsure whether your vision loss is due to XLRP and genetic testing has not been done, you can contact Serva Health at call 1-855-VIEWVISTA (855-843-9847) to speak directly with a nurse.

You may be asked to complete a pre-screening visit prior to screening for the clinical trial. This is an abbreviated visit that allows the clinical trial site to better understand your eligibility before completing the full screening requirements.

While there is no guarantee that this clinical trial will improve your XLRP symptoms, what researchers learn may lead to better treatments for patients with XLRP in the future.

• You may have to travel for the study visits depending on your study site.
• All travel for study-related assessments will be covered by the sponsor at no cost to you.
• It is important to consider the time associated with study participation.

Beacon Therapeutics, the clinical trial sponsor, is an ophthalmic gene therapy company founded to restore and improve the vision of patients with both prevalent and rare retinal diseases that result in blindness.

Beacon Therapeutic’s lead development candidate is AGTC-501, a late-stage gene therapy program for the treatment of XLRP.

You can contact Serva Health at 1-855-VIEWVISTA (855-843-9847) to speak directly with a nurse.

If you still have questions about clinical trials, or clinical research in general, there are additional resources that can help!

Click here to learn more about clinical research and questions you should ask.