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Study on the Safety and Effectiveness of AGTC-501 for Men with X-linked Retinitis Pigmentosa

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What is this study about?

This clinical trial is focused on studying a genetic eye condition called X-linked retinitis pigmentosa, which is caused by mutations in the RPGR gene. This condition affects vision, often leading to a gradual loss of sight. The study is testing a new treatment called AGTC-501, which is a type of gene therapy. Gene therapy involves using a specially designed virus to deliver a healthy copy of the gene into the cells of the eye. In this trial, AGTC-501 is given as a single injection directly into the retina, the part of the eye that senses light.

The purpose of the study is to evaluate the safety and effectiveness of AGTC-501 in improving vision in participants with this condition. Participants will be divided into groups, with some receiving the treatment and others not receiving any treatment (untreated control group). The study will monitor changes in vision and eye health over time, using various tests to assess how well the treatment works. These tests include measuring the sensitivity of the retina to light and checking the structure of the eye.

The study will last for several years, with regular check-ups to track the progress of participants. The main goal is to see if AGTC-501 can improve vision and whether it is safe to use. Participants will be closely monitored for any side effects or changes in their condition. This research aims to provide new insights into treating X-linked retinitis pigmentosa and potentially offer a new option for those affected by this genetic eye disease.

1 joining the study

Upon joining the study, the participant will undergo a series of initial assessments to confirm eligibility. These assessments include tests of visual and retinal function and structure in both eyes.

The participant must have a clinical diagnosis of X-linked retinitis pigmentosa (XLRP) and meet specific criteria related to age, gender, and genetic markers.

2 baseline assessments

Baseline assessments will be conducted to measure the participant’s current visual sensitivity and retinal health. This includes macular integrity assessment (MAIA) microperimetry and spectral-domain optical coherence tomography (SD-OCT).

These assessments will help establish a reference point for evaluating changes over the course of the study.

3 administration of AGTC-501

The participant will receive a single subretinal injection of AGTC-501, which is an adeno-associated virus vector containing the human RPGR gene. This procedure is performed to potentially improve visual function.

The injection is administered under controlled conditions, and the participant will be monitored closely for any immediate reactions.

4 post-injection monitoring

Following the injection, the participant will be monitored for safety and any adverse effects. This includes regular check-ups and assessments to ensure the participant’s well-being.

The participant may receive medications such as prednisolone or prednisone to manage inflammation, administered orally as prescribed by the study team.

5 follow-up assessments

The participant will undergo follow-up assessments at various intervals, including months 12, 18, and 24, to evaluate changes in visual sensitivity and retinal health.

These assessments will include MAIA microperimetry, mobility tests, and other evaluations to measure the efficacy of the treatment.

6 completion of the study

Upon completion of the study, the participant will have a final assessment to determine the overall impact of the treatment on their visual function.

The study aims to conclude by September 2030, with all data collected and analyzed to assess the safety and efficacy of the treatment.

Who Can Join the Study?

  • Provide written informed consent or assent, which means agreeing to participate in the study after understanding what it involves. If the participant is underage, a parent or guardian must also provide consent.
  • Be able to perform all tests related to vision and eye health in both eyes, as determined by the study doctor.
  • Have a certain level of macular sensitivity, which is the ability to see details in the center of the vision, measured by a specific test called MAIA microperimetry.
  • Have a detectable sub-foveal ellipsoid zone (EZ) line, which is a specific part of the retina, as seen in an eye scan called SD-OCT.
  • If both eyes qualify, the study will focus on the eye with worse vision, unless both eyes have the same vision level, in which case the study doctor will decide.
  • Be between 12 and 50 years old at the time of giving consent.
  • Be male and have a specific genetic change in the RPGR gene, which is related to the condition being studied.
  • Have a clinical diagnosis of X-linked retinitis pigmentosa (XLRP), which is the condition being studied.
  • Be in good general health to safely undergo eye surgery and take medications needed for the study.
  • Be able and willing to follow study instructions, complete assessments, and attend all study visits.
  • If the participant has a parent or caregiver, they must also be able to follow study instructions and attend visits with the participant.
  • Have a certain level of vision, measured by reading letters on an eye chart, with specific scores required for participation.
  • Have a certain level of low-light vision, also measured by reading letters on an eye chart, with specific scores required for participation.

Who Cannot Join the Study?

  • Only males can participate in the study. Females are not eligible.
  • Participants must have a specific eye condition called X-linked retinitis pigmentosa (XLRP). This is a genetic disorder that affects vision.
  • Participants must be within certain age ranges, which are not specified here.
  • Individuals who are considered part of a vulnerable population are not eligible. This term refers to groups of people who might be at a higher risk of harm or exploitation.

Where you can join this trial?

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Other Sites

Site Name City Country Status
Centro Medico Teknon-Grupo Quironsalud Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Spain Spain
Not yet recruiting
23.09.2024

Trial locations

Investigated drugs:

AGTC-501 is an investigational gene therapy being studied for its potential to treat X-linked Retinitis Pigmentosa, a genetic condition that affects vision. The therapy is administered through a single subretinal injection, which means it is delivered directly under the retina. The study aims to evaluate how effective, safe, and tolerable this treatment is for male participants with this condition.

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 characterized by the progressive loss of vision, starting with night blindness and loss of peripheral vision. Over time, individuals may experience a narrowing of the visual field, often described as “tunnel vision.” The condition is caused by mutations in genes located on the X chromosome, which is why it predominantly affects males. As the disease progresses, it can lead to significant visual impairment or even blindness. The rate of progression and severity can vary among individuals.

Trial ID:
2024-511181-36-00
Protocol code:
AGTC-RPGR-002
NCT ID:
NCT04850118
Trial Phase:
Therapeutic confirmatory (Phase III)

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