Retinitis pigmentosa describes a group of inherited eye diseases that damage the light-sensing cells in the retina. It typically starts with night blindness and loss of side vision, progressing over years or decades. About 1 in 4,000 people have RP, and until recently no treatments existed.
What's actually going on in research
Trials are testing gene therapies for specific mutations, optogenetic therapies that make remaining retinal cells light-sensitive, and retinal implants that bypass damaged cells. Researchers are also studying neuroprotective drugs to slow cell death, stem cell approaches to replace lost photoreceptors, and CRISPR-based gene editing.
Gene therapies
Voretigene neparvovec became the first FDA-approved gene therapy for RPE65-mutation RP in 2017. Trials are now testing gene therapies for other mutations, including those in the RHO and RPGR genes.
Optogenetics
These therapies use light-sensitive proteins to make surviving retinal cells responsive to light. Early trials show some people regain the ability to detect objects and navigate spaces.
CRISPR editing
Trials are testing whether CRISPR can directly edit mutations in the eye. This approach could work for dominant mutations where removing the faulty gene copy might preserve vision.
What to know before you search
Eligibility depends on the specific gene mutation, amount of remaining vision, disease stage, and whether the retina still has enough cells to respond to treatment.
What types of trials are currently open
- Gene therapy trials — Testing one-time injections that deliver working copies of specific genes into retinal cells. Each therapy targets a particular mutation.
- Optogenetic trials — Testing therapies that make remaining retinal cells light-sensitive, paired with special goggles that amplify light patterns.
- Neuroprotection trials — Testing drugs or devices that aim to keep photoreceptor cells alive longer, regardless of the underlying mutation.
- Retinal implant trials — Testing electronic devices implanted in the eye to bypass damaged cells and stimulate the optic nerve directly.
- Natural history studies — Following people with RP over time to measure how vision changes and identify factors that predict faster progression.
Recently added Retinitis Pigmentosa trials
Take a new obesity medication for rare genetic conditions
Bardet-Biedl syndrome (BBS) and other rare disorders associated with impairment of the melanocortin-4 receptor (MC4R) pathway are characterized by severe early-onset obesity, hyperphagia, and substantial morbidity. Setmelanotide, an MC4R agonist, is approved in Europe for selected genetic obesity disorders and reimbursed in Germany for eligible patients. This study aims to evaluate the effectiveness, safety, treatment persistence, metabolic outcomes, and patient-reported outcomes of Setmelanotide under real-world conditions. The registry is designed to allow future inclusion of additional MC4R agonists as they become approved and clinically available. The study will primarily be conducted at University Hospital Essen and will collect longitudinal routine clinical data from pediatric and adult patients receiving MC4R agonist therapy according to approved indications.
Track vision changes while receiving electrical stimulation therapy
Retinitis pigmentosa and similar degenerative diseases of the retina lead to progressive loss of vision. TES therapy with the CE-marked OkuStim® System is a treatment approved in the EU for slowing the progression of the disease. Patients increasingly report short-term subjective improvements in vision, which have not yet been systematically investigated. This exploratory study is conducted to determine whether these subjective short-term effects can be measured, and therefore also be quantified, by objective tests.
Find Retinitis Pigmentosa trials matched specifically to you
Answer 3 quick questions and we'll show you trials that fit your situation.