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Learn About Clinical Studies

What Is XLRP?

  • 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.

What Is a Clinical Trial?

  • 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.

What is laru-zova (AGTC-501) 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.

What if I have not had genetic testing?

  • 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.

Why am I being asked to pre-screen?

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

Why participate in laru-zova trials?

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.

Who is the sponsor?

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.

What if I still have questions about the laru-zova trials?

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

What if I still have questions about clinical trials?

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.

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Source: National Library of Medicine, “Learn about Studies,” available at https://clinicaltrials.gov/ct2/about-studies/learn#Considerations, This page last reviewed on May 24, 2023. Accessed February 9, 2024.