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 investigational study drug (laru-zova, also named 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.

An investigational drug is a therapy not yet approved by the country’s regulatory agency for use in humans or as a treatment for a particular disease or condition outside of a research setting.

The LANDSCAPE 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 LANDSCAPE trial if:

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

• If eligibility criteria are met, participants will then attend 3 screening visits at a study site.
• Eligible participants will undergo surgery to receive an injection of laru-zova (also named AGTC-501) in both eyes.

• Trial participation will last up to 5 years.

• If you have been diagnosed with XLRP and have had the RPGR gene mutation confirmed through genetic testing, you may be eligible for the LANDSCAPE 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 1-855-843-9847 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 laru-zova (also named AGTC-501), a late-stage gene therapy program for the treatment of XLRP.

You can contact Serva Health at 1-855-843-9847 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.