Retinal Dystrophies

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17 Retinal Dystrophies Trials Near You

Power is an online platform that helps thousands of Retinal Dystrophies patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

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No Placebo
Highly Paid
Stay on Current Meds
Pivotal Trials (Near Approval)
Breakthrough Medication

ACDN-01 for Stargardt Disease

Cincinnati, Ohio
This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2

13 Participants Needed

VP-001 for Retinal Dystrophy

Ann Arbor, Michigan
This is a repeat-dose, open-label, four arm safety and efficacy study of two doses of VP-001 administered intravitreally in participants with confirmed PRPF31 mutation-associated Retinal Dystrophy and previously treated with VP001.
No Placebo Group

Trial Details

Trial Status:Not Yet Recruiting
Trial Phase:Phase 1, 2

12 Participants Needed

VP-001 for Retinal Dystrophy

Ann Arbor, Michigan
A Phase 1 Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1
Age:12+

12 Participants Needed

Oral Metformin for Stargardt Disease

Ann Arbor, Michigan
Background: ABCA4 retinopathy is a genetic disease in which the ABCA4 protein is absent or faulty. It can cause waste material to collect in the eye and may cause cells to die. The cell death can lead to vision loss. Researchers want to see if an oral drug called metformin can help. Objective: To see if metformin is safe and possibly helps to slow the rate of ABCA4 retinopathy. Eligibility: People age 12 and older who have ABCA4 retinopathy and have problems with their vision. Design: Participants will be screened under a separate protocol. Participants will have a medical and family history. They will complete a questionnaire about their vision and daily activities. They will have a physical exam. They may have blood drawn through a needle in the arm. Participants will have an eye exam. Their pupils may be dilated with eye drops. Their retina may be photographed. Participants will have a visual field test. They will sit in front of a large dome and press a button when they see a light within the dome. Participants will have an electroretinogram. It examines the function of the retina. They will sit in the dark for 30 minutes. Then their eyes will be numbed with eye drops. They will wear contact lenses that can sense signals from the retinas. They will watch flashing lights. Participants will have optical coherence tomography. This non-invasive procedure makes pictures of the retina. Participants will have fundus autofluorescence. A bright blue light will be shone into their eye. Participants will take metformin by mouth for 24 months. Participants will have study visits every 6 months. Participation will last for at least 36 months....
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:12 - 100

56 Participants Needed

VP-001 for Retinal Dystrophy

Ann Arbor, Michigan
This trial tests a new eye injection treatment called VP-001 for people with a genetic condition that affects their vision. The goal is to see if it is safe and can help the damaged cells in their eyes.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1

20 Participants Needed

Gene Therapy for Leber Congenital Amaurosis

Ann Arbor, Michigan
The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A\>G in intron 26 of the CEP290 gene ("LCA10-IVS26").
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:3+

34 Participants Needed

Gene Therapy with SPVN06 for Cone-Rod Dystrophy

Pittsburgh, Pennsylvania
This trial is testing a new treatment for patients with advanced vision problems caused by specific genetic mutations. The study aims to determine the appropriate dose and evaluate its effectiveness.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2

33 Participants Needed

Natural History Study for Retinitis Pigmentosa

Pittsburgh, Pennsylvania
This is natural history study of rods and cones degenerations in patients with Retinitis Pigmentosa (RP) caused by pathogenic mutations in RHO, PDE6a or PDE6b gene mutations.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Unphased

82 Participants Needed

Gene Therapy with Light-Stimulating Glasses for Retinitis Pigmentosa

Pittsburgh, Pennsylvania
This trial is testing a new gene therapy injected into the eye and special light-stimulating glasses for patients with a specific type of vision loss called non-syndromic Retinitis Pigmentosa. The gene therapy aims to fix genetic problems in the eye, while the glasses help activate the treated cells. Gene therapy has shown promise in treating retinal diseases.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2

15 Participants Needed

DF-003 for ROSAH Syndrome

Bethesda, Maryland
The purpose of this study is to evaluate the safety and tolerability of DF-003 in retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache (ROSAH) syndrome patients.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 65

12 Participants Needed

VR Assessment for Retinal Disease

Bethesda, Maryland
Background: The retina is a thin layer of tissue at the back of the eye. Retinal disease usually reduces a person s mobility because it affects how he or she moves through familiar and unfamiliar environments. Researchers want to see if a virtual reality (VR) tool can provide an easier and more accurate way to assess mobility. Objective: To learn if researchers can track changes in mobility in people with retinal disease using a new VR tool. Eligibility: People aged 5 and older with retinal disease that affects their vision, and healthy volunteers. Design: Participants will have 2-3 clinic visits. Participants will wear goggles or sit in front of a screen while sitting. Using a game controller, they will navigate through 4 obstacle courses presented in VR. Participants will have a medical history exam. They will answer questions about their family history. They will fill out questionnaires about the vision and mobility issues they have in their daily lives. Participants will have a complete eye exam. They will read letters from a chart. Their eye pressure will be measured. Their pupils may be dilated with eye drops. Pictures of their eye will be taken. Lights will be shined in their eyes. Participants will take a visual field test. For this, they will look into a dome and press a button when they see a light. Participants will have an electroretinogram. For this, they will sit in the dark with their eyes patched. Then their eyes will be numbed with eye drops and they will wear contact lenses while watching flashing lights. Participants will have optical coherence tomography. This is a noninvasive procedure. It produces cross-sectional pictures of the retina....
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1
Age:5+

165 Participants Needed

Imaging for Retinal Dystrophy

Durham, North Carolina
The goal of this observational study is to utilize a novel imaging system designed for high-resolution retinal imaging of neonates, infants and children to identify the signs of photoreceptor development and degeneration in children with early-onset inherited retinal dystrophies (EORDs). Participants will have research imaging with SS-HH-OCT at the time of clinically-indicated eye examinations or procedures. The investigators aim to establish the basis for utilization of OCT imaging in earlier diagnosis and disease monitoring in children with EORDs. This work will set data reference standards and IRD endpoints that can be used in clinical trials.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased
Age:0 - 8

80 Participants Needed

Disulfiram for Retinal Degeneration

Rochester, New York
Oral disulfiram (Antabuse®) has been shown to improve image-forming vision in animal models with retinal degeneration due to its ability to decrease Retinoic Acid synthesis and consequently reduce hyperactivity in the inner retina. The investigator will aim to evaluate the impact of oral disulfiram on the vision of patients with retinal degeneration who are being treated with the drug in the management of their concurrent alcohol use disorder.
No Placebo Group

Trial Details

Trial Status:Not Yet Recruiting
Trial Phase:Phase 1

15 Participants Needed

OpCT-001 for Retinitis Pigmentosa

Madison, Wisconsin
Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 will focus on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2

54 Participants Needed

Gene Therapy for Leber Congenital Amaurosis

Philadelphia, Pennsylvania
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl). At least twenty-four subjects, three years of age or older, will be recruited. The intervention group will receive AAV2-hRPE65v2 at either The Children's Hospital of Philadelphia or University of Iowa to determine if it improves visual and retinal function in individuals with RPE65 gene mutations.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:3+

31 Participants Needed

Stem Cell Therapy for Retinal and Optic Nerve Disorders

Westport, Connecticut
This trial will use stem cells from patients' own bone marrow to treat severe vision loss caused by retinal or optic nerve damage. The stem cells will be injected into the eye to help repair the damaged tissues. SCOTS is the largest ophthalmology stem cell study registered at the National Institutes of Health, using autologous bone marrow-derived stem cells for retinal and optic nerve diseases.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased

500 Participants Needed

Disulfiram for Retinal Degeneration

Seattle, Washington
Aberrant retinoic acid signaling driven by the degenerating outer retina leads to pathological changes to the inner retina. The resulting hyperactivity of retinal ganglion cells leads to further diminution of the remaining vision in those afflicted with inherited retinal diseases. Inhibition of this pathway has led to improved visual function in murine models of retinal degeneration. This can be accomplished in humans with the FDA-approved irreversible inhibitor of aldehyde dehydrogenases, disulfiram.

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2

30 Participants Needed

Why Other Patients Applied

"My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."

HZ
Arthritis PatientAge: 78

"I've tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."

ZS
Depression PatientAge: 51

"I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."

WR
Obesity PatientAge: 58

"I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."

FF
ADHD PatientAge: 31

"As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."

IZ
Healthy Volunteer PatientAge: 38

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Bask GillCEO at Power
Learn More About Trials
How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

Frequently Asked Questions

How much do Retinal Dystrophies clinical trials pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do Retinal Dystrophies clinical trials work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Retinal Dystrophies trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Retinal Dystrophies is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a Retinal Dystrophies medical study?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest Retinal Dystrophies clinical trials?

Most recently, we added VP-001 for Retinal Dystrophy, Disulfiram for Retinal Degeneration and DF-003 for ROSAH Syndrome to the Power online platform.

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