Treatment for Retinitis Pigmentosa

Recruiting · 18+ · All Sexes · London, United Kingdom

This study is evaluating whether a gene therapy may help treat Retinitis Pigmentosa.

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About the trial for Retinitis Pigmentosa

Eligible Conditions
Non-syndromic Retinitis Pigmentosa · Retinitis · Retinitis Pigmentosa

Treatment Groups

This trial involves a single treatment. Treatment is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.


This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Retinitis Pigmentosa or one of the other 2 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Age 18 to 75 years old when the ICF was signed. show original
The patient has a history of vision problems, and mid-peripheral vision is currently impaired show original
The DSMB is still reviewing data from the dose-escalation cohort, so there is no better visual acuity information for the extension cohort at this time. show original
Refractive error of the study eye was within the range from -6 diopters to +6 diopters. show original
The main non-selection criterion is that the candidate is not a member of the Communist Party of the Soviet Union show original
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Odds of Eligibility
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Week 52/Year 1
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Week 52/Year 1.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Treatment will improve 1 primary outcome and 5 secondary outcomes in patients with Retinitis Pigmentosa. Measurement will happen over the course of Week 52/Year 1.

Evaluate the treatment effect of GS030 as assessed by mobility.
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with line task (before and after gene transfer, with GS030-MD turned ON and turned OFF).
Evaluate the treatment effect of GS030 as assessed by visual acuity
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with the Freiburg Visual Acuity & Contrast Test (FrACT) free computer program that uses graphics capabilities and psychometric methods to provide automated, self-paced measurement and the full field threshold stimulus test (FST) measuring the illuminance necessary to stimulate the most sensitive parts of the retina, and thus determines a quantifiable stimulation threshold (before and after gene transfer, with GS030-MD turned ON and turned OFF).
The safety and tolerability of escalating doses of GS030-DP administered via a single IVT and repeated light stimulation using GS030-MD in subjects with non-syndromic Retinitis Pigmentosa
Safety and tolerability of GS030 treatment at Week 52/Year 1, by assessments based on local and systemic safety issues, specifically those related to IVT of GS030-DP and the subsequent repeated use of GS030-MD, as assessed by incidence of Adverse Events.
Evaluate immune response to recombinant adeno associated viral vector, derived from serotype 2 (rAAV2.7m8) and ChR tdT protein.
Immune response to rAAV2.7m8 and ChR-tdT protein from baseline to week 52
Evaluate the treatment effect of GS030 as assessed by QoL
Assessment of the treatment effect on quality of life changes from baseline to Week 52 with the Short Form Survey 36 Version 2 (SF-36v2). The SF-36v2 is a subject-rated 36-item questionnaire assessing subject health. There are 8 scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0 to 100 scale. A lower score indicates more disability, and a higher score indicates less disability (a score of 0 is equivalent to maximum disability, and a score of 100 is equivalent to no disability).
Evaluate the treatment effect of GS030 as assessed by visual function
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Humphrey visual field 10-2 Standardized automated perimetry, square localization test, displaying white square at a random location on a black background and direction of motion test measuring the ability of subjects to determine the direction of an object moving in the visual field (before and after gene transfer, with GS030-MD turned ON and turned OFF).

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are common treatments for retinitis pigmentosa?

Although the current standard of care for RP is gene therapy, more options exist. Several treatments are currently under investigation, including cell replacement, laser therapy and other therapies.

Anonymous Patient Answer

Can retinitis pigmentosa be cured?

A cure can be reached. There is a chance and it is possible by using appropriate techniques to stop retinitis pigmentosa progressing and prevent further blindness.

Anonymous Patient Answer

What causes retinitis pigmentosa?

There are three types of RP: 1) autosomal dominant RP, 2) autosomal recessive RP and 3) autosomal non-mendelian RP. In autosomal recessive RP, there is a high frequency of deletions in the RP 1 gene and the non-mendelian RP category contains patients with different mutations in the same gene. For autosomal non-mendelian RP, a deletion in the RP 2 gene was shown to be a major cause. The two genes can be mapped to Chromosome 16q.

Anonymous Patient Answer

What are the signs of retinitis pigmentosa?

Symptoms of an impaired vision include blurred vision and poor central vision. Symptoms of other eye abnormalities that may occur with retinitis pigmentosa include abnormal colours in the vision, excessive light sensitivity and a loss of colour discrimination. Other symptoms of retinitis pigmentosa that may occur include strabismus, nystagmus, floaters and floaters. The lack of colour is frequently associated with nystagmus.

Anonymous Patient Answer

What is retinitis pigmentosa?

If you've been diagnosed with RP, then you are probably well aware of the symptoms, which range from a gradual loss of vision that affects both eyes, to severe blinding of one eye and double vision.\n\nThe word "Retinitis" comes from "retin-," (from the Greek alpha "a-ret-in'" "clear or bright [eye];" New Latin masculine gender noun Retina, from Greek alpha "a-ret-en'" "clear or bright [eye];" ultimately from Ancient Greek "a-rhein" "to shine," in references to ocular light\n\n- GeneReviews\nGeneReviews.

Anonymous Patient Answer

How many people get retinitis pigmentosa a year in the United States?

The U.S. retinitis pigmentosa patient population is small but growing, with 1-2 patients per 10,000 births annually. Most patients will manifest their disease before the age of 20 years, so that, by the year 2030, the U.S. RP patient population will be the largest in the world.

Anonymous Patient Answer

Have there been any new discoveries for treating retinitis pigmentosa?

No advancements have been made in treatments for retinitis pigmentosa. The only current therapies are to improve vision for affected patients, such as laser vision enhancement. This can be achieved in varying degrees, but it is not a cure for the disease. Future therapies may be developed for the disease, such as gene therapy, which can fix certain genes that cause retinsitis pigmentosa by inserting normal genes into the genetic background. This treatment could potentially cure up to 100% of the affected gene sets for retinitis pigmentosa and possibly other retinal disorders. There is also new research that is looking into stem cells, as it is proven to help stop retinitis pigmentosa in animal modeling.

Anonymous Patient Answer

What are the common side effects of treatment?

Treatments can cause nausea, vomiting, and diarrhea. In a study of people with retinitis pigmentosa, treatment with subretinal brimonidine reduced some of the common side effects related to treatment in this group. When they occur, all of these side effects are short-lived. There were no serious side effects in any study and none of the side effects in this study were permanent. The risks of the therapies are very small. Very few people have permanent or permanent severe side effects. The most common side effect is blurred vision, which is a rare side effect but can be temporary. Treatment of patients undergoing this type of surgery may worsen pre-existing eye disease, but only a small proportion of people have such an issue.

Anonymous Patient Answer

Does retinitis pigmentosa run in families?

Data from a recent study do not support the contention that ARRP-causing mutations are inherited in an autosomal dominant pattern with incomplete penetrance. Further investigation on this matter is needed.

Anonymous Patient Answer

How serious can retinitis pigmentosa be?

Because retinitis pigmentosa is rare, many patients with this eye disease are not aware of its seriousness. Some patients have vision for many years before they are aware of their ailment, and the impact on quality of life is significant. The most serious manifestation of retinitis pigmentosa is profound vision loss, which is extremely debilitating.

Anonymous Patient Answer

What is treatment?

This is the first study of patient-reported outcomes of retinal implants. A large percentage of patients with retinal implants experience significant vision loss and other visual symptoms. When patients perceive the potential for vision loss and vision recovery, they have a powerful motivation to continue with the therapy.

Anonymous Patient Answer

Does treatment improve quality of life for those with retinitis pigmentosa?

The use of amblyopia therapy resulted in a significant improvement in the quality of life and a lower QoL for those with RP in the short term.

Anonymous Patient Answer
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