Condition

Retinitis Pigmentosa clinical trials and research

• 29Clinical trials
• 39NIH research projects
• 21Countries

Newest Retinitis Pigmentosa clinical trials

All recruiting trials (29)

• Functional visual field and navigation testing for moderate-to-profound inherited retinal disease

  This project will test whether functional visual field and navigation tests can measure real-world vision and mobility in people with moderate-to-profound inherited retinal diseases.

  Recruiting Ray Therapeutics, Inc. · Irvine, California · NCT07502664

• OCT-980 oral treatment for rhodopsin-associated autosomal dominant retinitis pigmentosa

  This program will test whether the oral drug OCT-980 is safe, well tolerated, and shows signs of benefit in adults with RHO-associated autosomal dominant retinitis pigmentosa and in healthy volunteers.

  Recruiting at 2 sites Phase 1/2 Octant, Inc. · Portland, Oregon and 1 other locations · NCT07408232

• Course of EYS-associated retinitis pigmentosa in adults from Russia and former CIS

  We will follow adults with pathogenic EYS gene variants from Russia and former CIS for four years to try to find reliable vision and retinal measures for future treatments.

  Recruiting Sensor Technology for Deafblind · Moscow · NCT07228793

• ZVS203e eye gene-editing therapy for a specific RHO-related retinitis pigmentosa

  This trial tests a single subretinal injection of ZVS203e to see if it’s safe and can help a specific RP caused by an RHO mutation.

  Recruiting Phase 1/2 Chigenovo Co., Ltd · Beijing, Beijing Municipality · NCT06952842

• Testing IGT001 for treating Retinitis Pigmentosa

  This study is testing a new treatment called IGT001 for people with retinitis pigmentosa to see if it can help improve their vision and how safe it is to use.

  Recruiting Phase 1 Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine · Shanghai · NCT06936787

• Safety of a one-time DSP-3077 retinal sheet injection in adults with retinitis pigmentosa

  This will test whether a single subretinal injection of DSP-3077 retinal sheets is safe and well tolerated in adults with retinitis pigmentosa.

  Recruiting at 2 sites Phase 1/2 Sumitomo Pharma America, Inc. · Boston, Massachusetts and 1 other locations · NCT06891885

• Intravitreal KIO-301 for people with late-stage retinitis pigmentosa

  This trial will test whether injections of KIO-301 into both eyes are safe and can improve vision or light perception in adults with late-stage retinitis pigmentosa.

  Recruiting at 5 sites Phase 2 Kiora Pharmaceuticals, Inc. · Sydney, New South Wales and 4 other locations · NCT06628947

• Assessing vision measurement methods for patients with retinal degeneration

  This study is testing different ways to measure vision in people with retinal degeneration to see how their eyesight affects daily life and how it changes over time.

  Recruiting Ray Therapeutics, Inc. · Irvine, California · NCT06375239

• Disulfiram (Antabuse) for vision in retinal degeneration

  This study will test whether taking oral disulfiram (Antabuse) improves vision in adults with retinal degeneration who are also being treated for alcohol use disorder.

  Recruiting Phase 1 University of Rochester · Rochester, New York · NCT06319872

• Gene therapy for patients with retinitis pigmentosa

  This study is testing a new gene therapy called ZM-02 to see if it can improve vision for people with retinitis pigmentosa.

  Recruiting Early Phase 1 Augelux Therapeutics · Beijing, Beijing Municipality · NCT06292650

• Gene therapy for Retinitis Pigmentosa caused by CNGA1 mutations

  This study is testing a new gene therapy called VG901 to see if it is safe and can help people with Retinitis Pigmentosa caused by CNGA1 gene mutations.

  Recruiting Phase 1 VeonGen Therapeutics GmbH · Tübingen · NCT06291935

• Using stem cell-derived vesicles to treat retinitis pigmentosa

  This study is testing if injections of tiny particles from stem cells can help people with retinitis pigmentosa improve their vision and stay safe during treatment.

  Recruiting Phase 1/2 Mahidol University · Bangkok Noi, Bangkok · NCT06242379

• Evaluating a new treatment for retinitis pigmentosa

  This study is testing a new eye implant to see if it can help improve vision in people with retinitis pigmentosa.

  Recruiting Eye & ENT Hospital of Fudan University · Shanghai, Shanghai Municipality · NCT05853107

• Understanding the natural history of Best Vitelliform Macular Dystrophy

  This study looks at how Best Vitelliform Macular Dystrophy changes over time in people with the condition to help find better treatments in the future.

  Recruiting at 2 sites Columbia University · New York, New York and 2 other locations · NCT05809635

• Gene editing treatment for Retinitis Pigmentosa

  This study is testing a gene editing treatment called ZVS203e to see if it can safely help people with Retinitis Pigmentosa caused by a specific gene mutation.

  Recruiting Early Phase 1 Peking University Third Hospital · Beijing, Beijing Municipality · NCT05805007

• Registry and natural history study of retinal dystrophies linked to rare genetic variants

  This study is trying to track how inherited eye conditions linked to rare genetic changes affect people over time, while also creating a registry to help understand these conditions better.

  Recruiting at 35 sites Jaeb Center for Health Research · Little Rock, Arkansas and 35 other locations · NCT05589714

• Imaging of outer retinal diseases using adaptive optics

  This study is trying to take detailed pictures of the retina in people with outer retinal diseases to help find better ways to diagnose and understand these conditions.

  Recruiting at 2 sites Food and Drug Administration (FDA) · Bethesda, Maryland and 1 other locations · NCT05355415

• Using stem cells from umbilical cords to treat non-arteritic ischemic optic neuropathy

  This study is testing if using stem cells from umbilical cords can help people with non-arteritic ischemic optic neuropathy improve their vision and overall eye health.

  Recruiting at 2 sites Phase 1 The Foundation for Orthopaedics and Regenerative Medicine · Naples, Florida and 1 other locations · NCT05147701

• Gene therapy for patients with retinitis pigmentosa

  This study is testing a new gene therapy to see if it can help people with retinitis pigmentosa who can only see light in one eye.

  Recruiting Phase 1/2 Bionic Sight LLC · Teaneck, New Jersey · NCT04278131

• Advanced imaging techniques for eye conditions

  This study is testing new advanced imaging techniques to see if they can help doctors better understand and diagnose eye diseases like retinitis pigmentosa and maculopathy.

  Recruiting Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts · Paris · NCT04129021

• Improving visual ability in patients with retinal prosthesis

  This study is testing new ways to improve vision for people with retinal prostheses by using special stimulation methods and training to help them see shapes better.

  Recruiting University of Michigan · Ann Arbor, Michigan · NCT03635645

• Using stem cells to treat retinal and optic nerve damage

  This study is testing if using stem cells from your own bone marrow can help improve vision in people with serious eye damage from conditions like age-related macular degeneration and optic nerve problems.

  Recruiting at 4 sites MD Stem Cells · Westport, Connecticut and 3 other locations · NCT03011541

• Investigating rod and cone function in retinal diseases

  This study is testing how well the light-sensitive cells in the eyes work in people with retinal diseases and healthy volunteers to help understand vision changes in low light.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT02617966

• Registry for inherited retinal degenerative diseases

  This study is creating a registry for people with rare inherited eye diseases to help track their health and experiences over time, making it easier to understand these conditions and support research.

  Recruiting Foundation Fighting Blindness · Columbia, Maryland · NCT02435940

• Evaluating pupil response to light in patients with retinal conditions

  This study is testing a new way to check vision by measuring how pupils respond to light in people with retinal conditions like glaucoma to see if it’s better than current methods.

  Recruiting Sheba Medical Center · Tel HaShomer · NCT02014389

• High-resolution imaging of the retina

  This study is testing a new way to take detailed pictures of the retina to help doctors better understand and treat eye diseases like Stargardt's and age-related macular degeneration in people aged 7 and older.

  Recruiting University of Pennsylvania · Philadelphia, Pennsylvania · NCT01866371

• Registry for patients with rare diseases

  This study is creating a central registry for people with rare diseases to help connect them with researchers and improve treatments.

  Recruiting Sanford Health · Sioux Falls, South Dakota · NCT01793168

• Collecting cells to study eye diseases

  This study is collecting hair, skin, and blood samples from people with certain eye diseases to see how these conditions develop and find new treatments.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT01432847

• High-resolution retinal imaging for patients with inherited retinal degenerations

  This study is testing advanced imaging techniques to see how inherited retinal diseases affect vision and to better understand the changes happening in the retina for people with conditions like retinitis pigmentosa and cone-rod dystrophy.

  Recruiting University of California, San Francisco · San Francisco, California · NCT00254605

NIH-funded research (39)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Inflammation in MERTK-related retinitis pigmentosa

  This project aims to understand whether losing the MERTK gene causes retinal inflammation that leads to vision loss in people with MERTK-related retinitis pigmentosa.

  NIH research University of Minnesota · Minneapolis, United States · NIH-11494188

• Enhancing cone survival in RP through targeted cell-specific CRISPR therapy

  A patient-friendly look at a new approach to protect cone cells in retinitis pigmentosa using precise gene editing to support vision.

  NIH research Columbia University Health Sciences · New York, United States · NIH-11420299

• Oral, once-daily MC16 medicine for retinitis pigmentosa

  A gene-mutation-agnostic pill (MC16) is being studied to help people with retinitis pigmentosa by addressing a common cellular problem in the eye.

  NIH research Mitochem Therapeutics INC. · Charleston, United States · NIH-11401308

• Role of IPM Proteoglycans in Retinal Health and Inherited Blindness

  This grant studies how specific retinal extracellular matrix proteins (IMPG1 and IMPG2) affect eye health and contribute to retinitis pigmentosa, with potential implications for people who have or are at risk for inherited blindness.

  NIH research West Virginia University · Morgantown, United States · NIH-11384637

• NAC Attack: a late-stage trial for preserving vision in RP patients

  A large, multi-site study is testing whether taking a daily NAC pill can help people with retinitis pigmentosa keep more of their vision.

  NIH research Johns Hopkins University · Baltimore, United States · NIH-11384455

• Non-invasive biomarker for mitochondria health in retinitis pigmentosa

  Aims to test a new, non-invasive OCT-based biomarker to explain and predict how mitochondria function affects cone cells in people with retinitis pigmentosa.

  NIH research Wayne State University · Detroit, United States · NIH-11365895

• Understanding and Treating Inherited Retinal Degeneration to Preserve Vision

  A clinician-scientist at Stanford is pursuing gene-agnostic therapies to slow or prevent vision loss from inherited retinal diseases like Retinitis Pigmentosa.

  NIH research Stanford University · Stanford, United States · NIH-11352439

• Sonogenetic Restoration of Vision for Retinitis Pigmentosa

  A new approach uses light-touch ultrasound to stimulate specific retinal cells so people with retinitis pigmentosa might regain better vision in the future.

  NIH research Nanoscope Technologies, LLC · Arlington, United States · NIH-11325386

• Finding new drugs to protect vision in zebrafish models of retinitis pigmentosa

  This study is looking for ways to help protect the eye cells that are damaged in retinitis pigmentosa, a condition that causes vision loss, by testing different drugs on specially modified zebrafish that act like this disease.

  NIH research University of Pittsburgh at Pittsburgh · Pittsburgh, United States · NIH-11314801

• Study of how RP2 mutations affect photoreceptor nerve cell development and vision

  Researchers are studying how genetic changes linked to X-linked retinitis pigmentosa disrupt special nerve-cell movements in the eye, which can lead to blindness in people with RP2 mutations.

  NIH research University of Michigan at Ann Arbor · Ann Arbor, United States · NIH-11312655

• Proteasome Boost to Protect Vision in Retinitis Pigmentosa

  A study to see if boosting the cell’s protein-cleanup machines can slow vision loss in people with retinitis pigmentosa.

  NIH research Duke University · Durham, United States · NIH-11312607

• N-acetylcysteine (NAC) to help preserve vision in retinitis pigmentosa

  This trial tests whether taking oral NAC daily can slow cone loss and help people with retinitis pigmentosa keep more vision.

  NIH research Johns Hopkins University · Baltimore, United States · NIH-11307042

• N‑acetylcysteine (NAC) to slow vision loss in retinitis pigmentosa with centralized OCT imaging support

  This project uses detailed OCT eye scans to see if N‑acetylcysteine (NAC) helps people with retinitis pigmentosa keep a key light‑sensing layer of their retina longer than a placebo.

  NIH research Duke University · Durham, United States · NIH-11306996

• Methotrexate and other medicines for rhodopsin-related retinitis pigmentosa

  This project tests whether methotrexate and similar repurposed drugs can protect vision in people with rhodopsin-linked autosomal dominant retinitis pigmentosa.

  NIH research University of Pittsburgh at Pittsburgh · Pittsburgh, United States · NIH-11295659

• Understanding how vision loss occurs in retinitis pigmentosa

  This study is looking at how vision loss happens in people with retinitis pigmentosa by using a special mouse model, and it hopes to find ways to help the brain adapt better to this condition, which could lead to new treatments to help preserve vision.

  NIH research University of Arizona · Tucson, United States · NIH-11267759

• One-vector gene therapy for dominant retinitis pigmentosa

  This gene therapy aims to lower harmful rhodopsin and replace it with a healthy copy to help people with autosomal-dominant retinitis pigmentosa caused by many RHO mutations.

  NIH research Opus Genetics INC. · Raleigh, United States · NIH-11187134

• Oral N‑acetylcysteine treatment for retinitis pigmentosa

  Adults with retinitis pigmentosa will take oral N‑acetylcysteine or a placebo to see whether it slows vision loss.

  NIH research Johns Hopkins University · Baltimore, United States · NIH-11179132

• How IMPG1 and IMPG2 proteins affect retinal health and retinitis pigmentosa

  This project looks at whether defects in two eye proteins (IMPG1 and IMPG2) disrupt nutrient flow between retinal cells, cause childhood- or adult-onset retinitis pigmentosa, and whether gene therapy can help.

  NIH research West Virginia University · Morgantown, United States · NIH-11176944

• Cell stress response to misfolded rhodopsin in retinitis pigmentosa

  This project looks at how cells cope with misfolded rhodopsin to help people with retinitis pigmentosa keep their vision.

  NIH research New York University School of Medicine · New York, United States · NIH-11169873

• A New Eye Scan to Understand and Help Retinitis Pigmentosa

  This project looks for a gentle way to measure eye health in people with retinitis pigmentosa, hoping to find new ways to help them.

  NIH research Wayne State University · Detroit, United States · NIH-11142437

• Understanding how certain retinal cells survive in a mouse model of Retinitis Pigmentosa

  This study is looking at how certain cells in the eye can stay healthy even when other cells are damaged in people with Retinitis Pigmentosa, and it hopes to find new ways to help protect vision for those affected by this condition.

  NIH research Massachusetts Eye and Ear Infirmary · Boston, United States · NIH-11127422

• Protecting cone vision in retinitis pigmentosa with targeted CRISPR therapy

  This project uses precise CRISPR gene editing to help cone photoreceptors survive in adults with retinitis pigmentosa.

  NIH research Columbia University Health Sciences · New York, United States · NIH-11124003

• Understanding how mutations affect the movement of photoreceptor cells in the retina.

  This study looks at how certain genetic changes affect the movement of important cells in the eye that help us see, and it aims to help people with inherited eye diseases understand more about their vision problems.

  NIH research University of Michigan at Ann Arbor · Ann Arbor, United States · NIH-11110329

• Using ultrasound to restore vision in patients with Retinitis Pigmentosa

  This study is looking at a new way to help people with Retinitis Pigmentosa regain some vision using ultrasound, which is a gentle sound wave technique, instead of surgery or gene therapy.

  NIH research Nanoscope Technologies, LLC · Arlington, United States · NIH-11077270

• Investigating how proteasome overload contributes to blindness from retinitis pigmentosa

  This study is looking at how a buildup of faulty proteins in the eye affects vision in people with retinitis pigmentosa, and it aims to find ways to help the eye cells clear these proteins to help protect sight for those with this condition.

  NIH research Duke University · Durham, United States · NIH-11073037

• Testing a treatment to help preserve vision in patients with retinitis pigmentosa

  This study is looking at whether taking N-acetylcysteine (NAC), a helpful antioxidant, can support vision in people with retinitis pigmentosa by protecting important cells in the eye, and participants will either receive NAC or a placebo over time to see how it affects their eyesight.

  NIH research Johns Hopkins University · Baltimore, United States · NIH-11045045

• Investigating the effects of N-acetyl cysteine on retinitis pigmentosa progression

  This study is looking at how a supplement called N-acetyl cysteine (NAC) might help slow down the eye disease retinitis pigmentosa by taking special pictures of your retina over time, and it’s for people who have this condition.

  NIH research Duke University · Durham, United States · NIH-11042760

• Understanding a specific genetic cause of retinal degeneration

  This study is looking at a specific type of retinitis pigmentosa called RP59, which is caused by changes in a gene, and researchers are using specially designed mice to learn more about how these changes affect vision, hoping to find new ways to help people with this condition.

  NIH research University of Alabama at Birmingham · Birmingham, United States · NIH-11042720

• Investigating inflammation in a type of inherited blindness

  This study is looking at how inflammation affects retinitis pigmentosa, a genetic eye condition that can cause vision loss, by using mice to see how a missing gene called MERTK impacts the eye's ability to clean up debris, with the hope of finding new treatment options for patients.

  NIH research Yale University · New Haven, United States · NIH-11017023

• Regeneration of light-sensing cells in a zebrafish model of blindness.

  This study is looking at how zebrafish can naturally heal their eye cells to help find new ways to restore vision for people with Retinitis Pigmentosa, a condition that causes blindness.

  NIH research University of Texas Hlth Sci Ctr Houston · Houston, United States · NIH-11010328

• Understanding how the brain preserves vision in retinitis pigmentosa

  This grant studies why people with retinitis pigmentosa keep some visual sharpness even as their eye cells fail, by looking at brain and eye circuits in mice and figuring out if changing plasticity could slow vision loss.

  NIH research University of Arizona · Tucson, United States · NIH-11000382

• Investigating how specific proteins in the eye affect retinal health and diseases like retinitis pigmentosa.

  This study is looking at how two proteins, IMPG1 and IMPG2, help keep the retina healthy and how problems with these proteins might lead to retinitis pigmentosa, a condition that can cause vision loss, especially in children, and it aims to find ways to treat these issues with gene therapy.

  NIH research West Virginia University · Morgantown, United States · NIH-10945359

• Finding a new way to measure energy in the eyes of patients with retinitis pigmentosa

  This study is looking at how we can better understand and treat retinitis pigmentosa by using a special imaging technique to see how well the cells in your retina are working, which could help doctors create more personalized treatment plans for you.

  NIH research Wayne State University · Detroit, United States · NIH-10931516

• Gene therapy for treating a common inherited eye disease.

  This study is testing a new gene therapy for people with autosomal-dominant retinitis pigmentosa, a genetic eye condition that causes vision loss, by using a special delivery method to fix the faulty gene and help improve their sight.

  NIH research Opus Genetics INC. · Raleigh, United States · NIH-10919095

• Improving cone cell survival in retinitis pigmentosa by altering metabolism

  This study is looking at ways to help protect the vision of people with retinitis pigmentosa by changing how certain cells in the eye get their energy, using special techniques in mice to find new treatments that could prevent blindness.

  NIH research Columbia University Health Sciences · New York, United States · NIH-10900808

• Understanding inner retinal changes in retinitis pigmentosa

  This study is looking at how retinitis pigmentosa affects the inner part of the eye after the light-sensing cells are lost, using special tests to see how this impacts vision, and it aims to find ways to help improve sight for people with this condition.

  NIH research University of Illinois at Chicago · Chicago, UNITED STATES · NIH-10896158

• Testing a new treatment for retinitis pigmentosa using N-acetylcysteine

  This study is looking at whether a supplement called N-acetylcysteine (NAC) can help slow down vision loss in people with retinitis pigmentosa, and if you join, you’ll either get NAC or a placebo for about three and a half years to see how it works.

  NIH research Johns Hopkins University · Baltimore, United States · NIH-10868502

• Finding new causes of retinitis pigmentosa using metabolic and protein analysis

  This study is looking at how retinitis pigmentosa affects the eyes at a molecular level in mice, hoping to find clues that could lead to new treatments for people with this condition before they experience serious vision loss.

  NIH research Univ of North Carolina Chapel Hill · Chapel Hill, United States · NIH-10694148

• Gene therapy for a type of inherited vision loss

  This study is working on a new gene therapy to help people with a specific type of vision loss called autosomal recessive retinitis pigmentosa, which is caused by changes in the CNGB1 gene, by using a special virus to deliver the correct gene to the eye and hopefully improve vision and protect the eye from further dama

  NIH research Michigan State University · East Lansing, United States · NIH-10368093

Related topics

Quick answers

How many Retinitis Pigmentosa clinical trials are recruiting right now?

29, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 21 countries. The most sites are in United States (18 trials), China (5 trials), France (3 trials), Australia (2 trials), Israel (2 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1/2: 5, Phase 1: 4, Early Phase 1: 2, Phase 2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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