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Long-Term Safety and Efficacy Study of Botaretigene Sparoparvovec for Patients with X-Linked Retinitis Pigmentosa

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called X-linked Retinitis Pigmentosa, which is a genetic disorder that leads to a gradual loss of vision. This condition is caused by mutations in the RPGR gene. The study aims to evaluate the long-term safety and effectiveness of a gene therapy treatment known as AAV5-hRKp.RPGR. This treatment involves delivering a modified virus to the retina, the part of the eye that senses light, to help improve vision in individuals affected by this genetic condition.

Participants in this study will receive the gene therapy treatment through a procedure called subretinal injection, where the solution is injected under the retina. The study will monitor the participants over an extended period to assess how well the treatment works and to ensure it is safe. The main focus will be on changes in vision and any side effects that may occur. The study will also involve the use of other medications, such as Omeprazole, which may be recommended for participants with certain risk factors, and Cefazolin Sodium, Vancomycin, and Cefuroxime, which are antibiotics used to prevent infections during eye surgeries. Additionally, Triamcinolone Acetonide, Dexamethasone Acetate, and Betamethasone are corticosteroids that may be used to reduce inflammation after surgery.

The purpose of this study is to gather information on how well the gene therapy works in improving vision and to monitor any potential side effects over time. Participants will be followed up regularly to check their vision and overall health. This study is part of a larger effort to find effective treatments for genetic eye conditions and to improve the quality of life for those affected by Retinitis Pigmentosa.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after completing a previous study, MGT-RPGR-021.

    Understanding the study's purpose and procedures is required.

  2. Step 2

    Initial assessment

    An initial assessment of vision is conducted to establish a baseline for future comparisons.

    This assessment includes a vision-guided mobility test.

  3. Step 3

    Treatment administration

    The treatment involves the delivery of AAV5-hRKp.RPGR through a subretinal injection.

    This procedure is designed to address vision issues related to X-linked Retinitis Pigmentosa.

  4. Step 4

    Follow-up assessments

    Regular follow-up assessments are conducted to monitor vision changes and treatment effects.

    These assessments include repeated vision-guided mobility tests.

  5. Step 5

    Monitoring for side effects

    Monitoring for any adverse effects is ongoing throughout the study.

    Laboratory assessments are performed to ensure safety and tolerability.

  6. Step 6

    Long-term evaluation

    The study aims to evaluate the long-term safety and effectiveness of the treatment.

    The study is expected to continue until December 19, 2029.

Who can join the trial?

4 criteria

  • The patient must have previously completed participation in Study MGT-RPGR-021.
  • The patient must understand the purpose of the study and the procedures involved, and be willing to participate.
  • The study is open to both male and female participants.
  • The study includes individuals from vulnerable populations, which means people who might need extra care or protection.

Who cannot join the trial?

8 criteria

  • Patients who do not have X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene cannot participate. This is a specific type of eye condition.
  • Individuals who are not within the specified age range for the study are excluded. The age range is not specified here, but it is important for eligibility.
  • Participants who are not able to provide informed consent or do not have a legal representative to do so on their behalf are excluded. Informed consent means understanding the study and agreeing to participate.
  • Patients with other medical conditions that might interfere with the study or its results are not eligible. This means if you have other health issues that could affect the study, you might not be able to join.
  • Individuals who have participated in other clinical trials recently may be excluded. This is to ensure that other treatments do not affect the study results.
  • Pregnant or breastfeeding women are typically excluded from clinical trials to protect the health of the mother and child.
  • Patients who have allergies or reactions to the study medication or its components cannot participate. This is to prevent any harmful reactions.
  • Individuals who are unable to follow the study procedures or attend required visits are excluded. This means if you cannot commit to the study schedule, you might not be eligible.
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Investigated drugs

AAV5-hRKp.RPGR is a gene therapy being studied for its potential to treat X-linked Retinitis Pigmentosa, a genetic disorder that affects vision. This therapy uses a modified virus to deliver a healthy copy of the RPGR gene to the patient's retinal cells. The goal is to improve or stabilize vision by addressing the underlying genetic cause of the disease.

What is already known about the treatment

AAV5-hRKp.RPGR – This medication is administered through an injection directly into the eye, specifically targeting the retina. It is currently in a Phase 3 clinical trial, focusing on its long-term safety and effectiveness for treating X-linked Retinitis Pigmentosa caused by mutations in the RPGR gene. The main therapeutic indication is to improve functional vision in affected individuals. At the molecular level, it works by delivering a healthy copy of the RPGR gene to retinal cells using a viral vector, which helps restore normal protein function and slow disease progression. It is classified as a gene therapy product.

Investigated diseases

X-Linked Retinitis Pigmentosa – This is a genetic disorder that affects the retina, the light-sensitive tissue at the back of the eye. It is caused by mutations in the RPGR gene and is inherited in an X-linked manner, meaning it primarily affects males. The disease leads to progressive vision loss, starting with difficulty seeing in low light conditions and loss of peripheral vision. Over time, individuals may experience a narrowing of the visual field, often described as "tunnel vision." As the condition progresses, it can lead to significant vision impairment. The rate of progression can vary among individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-511411-25-00Protocol codeMGT-RPGR-022Estimated enrolment95 patients

sourced from the EU Clinical Trials Register and site verification

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