Treatment for Retinitis Pigmentosa
This study is evaluating whether a gene therapy may help treat Retinitis Pigmentosa.
See full descriptionAbout the trial for 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.
Eligibility
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.
Approximate Timelines
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.
Patient Q & A Section
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.