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Study of GS030 gene therapy safety and tolerability in patients with Retinitis Pigmentosa

InvestigationalNo 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 focuses on testing a treatment for Retinitis Pigmentosa, a genetic eye disease that causes gradual loss of vision. The study evaluates a new gene therapy called GS030-DP, which is given as a single injection into the eye, combined with the use of special light-stimulating equipment called GS030-MD.

The treatment involves an adeno-associated viral vector, which is a modified virus that carries genetic material to help restore light sensitivity in the eye. The medication is administered through an intravitreal injection, which means it is injected directly into the eye. After the injection, patients use special light-stimulating equipment as part of the treatment.

The main goal of this study is to test different doses of the treatment to determine if it is safe and well-tolerated by patients. The study will monitor patients for one year after receiving the treatment to check for any side effects and evaluate how well the treatment works. During this time, various tests will be performed to measure changes in vision and eye structure.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial treatment

    You will receive a single intravitreal injection (an injection into the eye) of GS030-DP medication.

    The treatment will be administered to one eye only.

    The dose will be determined based on your assigned group in the study.

  2. Step 2

    Light stimulation device usage

    You will be provided with a special device called GS030-MD that produces light stimulation.

    You will need to use this device regularly throughout the study period.

    The medical team will provide instructions on proper usage and wearing of the device.

  3. Step 3

    Regular monitoring visits

    Your progress will be monitored for 52 weeks (1 year).

    During visits, the medical team will perform various vision tests including:

    - Tests for visual function and ability to see shapes

    - Eye imaging scans

    - Pictures of the back of your eye

    - Quality of life assessments using questionnaires

  4. Step 4

    Safety monitoring

    Throughout the study period, any side effects or reactions will be recorded.

    Both local effects in the eye and general health effects will be monitored.

    Regular eye examinations will be performed to check the health of your treated eye.

  5. Step 5

    Birth control requirements

    If applicable, you must use effective birth control methods for 12 months after receiving the eye injection.

    Female participants will need regular pregnancy tests during the study.

    Male participants must use appropriate contraception for 12 months after receiving the treatment.

Who can join the trial?

14 criteria

  • Must be between 18 and 75 years old when signing the consent form
  • Must sign an informed consent form
  • Must be able to wear and properly use the special light-stimulating device (GS030-MD)
  • Must have an interpupillary distance (distance between the centers of the pupils) between 51 mm and 72 mm
  • Must have a refractive error (vision correction need) between -9 and +6 diopters in the study eye
  • Must be able to tolerate repeated light stimulation from the device
  • Must have a diagnosis of non-syndromic retinitis pigmentosa confirmed by clinical examination or special eye tests
  • Must have visual acuity no better than light perception or counting fingers (depending on study group)
  • Must have relatively preserved inner retinal layers as measured by special eye imaging (SD-OCT)
  • Must retain memory of previous useful vision
  • Must be able to achieve adequate pupil dilation for eye examinations
  • Must test negative for HIV infection
  • For women of childbearing age: Must have negative pregnancy tests and agree to use effective birth control for 12 months after treatment
  • For men: Must agree to use condoms for 12 months after treatment

Who cannot join the trial?

14 criteria

  • Age below 18 years or above 75 years
  • Previous participation in any gene therapy clinical trials
  • Any active eye infection or inflammation
  • History of other significant eye diseases besides Retinitis Pigmentosa
  • Pregnancy or breastfeeding
  • Major surgery within 3 months before the study
  • Participation in another clinical trial within 30 days
  • Significant medical conditions that could interfere with the study, such as:
    • Uncontrolled high blood pressure
    • Unstable heart disease
    • Severe diabetes
    • Known allergies to study medications or similar compounds
    • Inability to comply with study procedures or follow-up visits
    • Use of medications that could interact with the study treatment
    • History of drug or alcohol abuse within the past year
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Investigated drugs

  • GS030-DP

    is an investigational therapy that is administered through a single injection into the eye (intravitreal injection). It is being studied for the treatment of retinitis pigmentosa, a genetic eye disease that causes progressive vision loss.

  • GS030-MD

    is a medical device component used in conjunction with GS030-DP. It provides light stimulation to the treated eye, working together with the injected therapy to potentially help improve vision in patients with retinitis pigmentosa.

What is already known about the treatment

  • GS030-DP

    An investigational gene therapy medication administered through intravitreal injection directly into the eye, specifically developed for treating retinitis pigmentosa, a genetic eye disorder that causes progressive vision loss. The therapy works by introducing light-sensitive proteins into retinal cells, aiming to restore some visual function in patients who have lost photoreceptor cells. This novel treatment is currently being evaluated in Phase 1/2a clinical trials to assess its safety and tolerability, and it represents a potential breakthrough in treating inherited retinal diseases.

  • GS030-MD

    A medical device component that works in conjunction with GS030-DP gene therapy, designed to provide controlled light stimulation to activate the treated retinal cells in patients with retinitis pigmentosa. The device is being tested as part of a combination therapy approach, where repeated light stimulation is used to activate the genetically modified retinal cells to potentially improve visual function. This innovative technology is currently under clinical investigation to determine its safety and effectiveness when used as part of the complete GS030 treatment system.

Investigated diseases

Retinitis Pigmentosa - A genetic eye disease that causes gradual degeneration of the retina, which is the light-sensitive tissue at the back of the eye. The condition typically begins with decreased night vision and loss of peripheral (side) vision in adolescence or young adulthood. As the disease progresses, it leads to the development of tunnel vision, where only central vision remains. The condition affects both eyes and is characterized by the presence of dark pigment deposits in the retina. The disease progression rate varies among individuals, with some experiencing a slower decline in vision than others.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-516059-42-00Protocol codeGS030_CLIN_001Estimated enrolment18 patientsSponsorGensight Biologics

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