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38 Retinitis Pigmentosa Trials near Miami, FL
Power is an online platform that helps thousands of Retinitis Pigmentosa 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.
Learn More About PowerGene Therapy for Retinitis Pigmentosa
Miami, Florida
This is a Phase 3 study to Assess the Efficacy, Safety and Tolerability of OCU400 in patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP.
This is a multicenter, assessor blinded and randomized study which will enroll 150 subjects.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:5+
Key Eligibility Criteria
Disqualifiers:Glaucoma, Retinal Detachment, Others
150 Participants Needed
N-acetylcysteine for Retinitis Pigmentosa
Miami, Florida
This trial is testing if N-acetylcysteine (NAC) can help slow down vision loss in people with Retinitis Pigmentosa (RP). RP is a genetic condition that causes gradual vision loss. NAC reduces damage caused by too much oxygen around eye cells, potentially protecting vision. N-acetylcysteine (NAC) has been shown to reduce oxidative damage and increase cone function/survival in studies related to RP.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18 - 65
Key Eligibility Criteria
Disqualifiers:Active Cancer, Renal Failure, Liver Disease, Others
483 Participants Needed
AGTC-501 for Retinitis Pigmentosa
Miami, Florida
This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec )) to an untreated control group in male participants with X-linked retinitis pigmentosa caused by RPGR mutations.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:12 - 50
Sex:Male
Key Eligibility Criteria
Disqualifiers:Herpes, Corticosteroid Allergy, Gene Therapy, Others
Must Not Be Taking:Anticoagulants, Corticosteroids
85 Participants Needed
VP-001 for Retinal Dystrophy
Miami, Florida
This is a Phase 1/2 repeat-dose, open-label, two-arm, parallel group safety and efficacy study of two doses of VP-001 (30 μg and 75 μg) in participants with confirmed PRPF31 mutation-associated retinal dystrophy, including participants previously treated with VP001 in the PLATYPUS Study or WALLABY Study for a minimum of 8 weeks.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Key Eligibility Criteria
Disqualifiers:Uncontrolled Systemic Disease, Other RP Mutations, Others
Must Not Be Taking:Anti-VEGF, Corticosteroids, Ozurdex
16 Participants Needed
Gene Therapy with SPVN06 for Cone-Rod Dystrophy
Miami, Florida
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
Key Eligibility Criteria
Disqualifiers:Gene Therapy, Cancer, Glaucoma, Others
Must Not Be Taking:Immunosuppressants, Corticosteroids, Others
33 Participants Needed
OCU400 for Retinitis Pigmentosa
Miami, Florida
This trial tests OCU400, an eye injection, in patients with specific genetic mutations causing RP and LCA. The treatment aims to correct faulty genes to prevent vision loss.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:6+
Key Eligibility Criteria
Disqualifiers:Gene Therapy, Intraocular Surgery, Infections, Others
22 Participants Needed
AGTC-501 for Retinitis Pigmentosa
Miami, Florida
This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 2
Age:12+
Sex:Male
Key Eligibility Criteria
Disqualifiers:Other Mutations, Pre-existing Eye Conditions, Others
24 Participants Needed
Laruparetigene Zovaparvovec for Retinitis Pigmentosa
Miami, Florida
The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:12 - 50
Sex:Male
Key Eligibility Criteria
Disqualifiers:Herpes, Immunosuppression, Autoimmunity, Others
Must Not Be Taking:Anticoagulants, Corticosteroids, Immunosuppressives, Others
10 Participants Needed
VP-001 for Retinal Dystrophy
Miami, Florida
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
Key Eligibility Criteria
Disqualifiers:Uncontrolled Systemic Disease, Other RP Mutations, Recent Ocular Surgery, Others
Must Not Be Taking:Anti-VEGF, Corticosteroid Injections
20 Participants Needed
BIIB111 for Choroideremia & BIIB112
Miami, Florida
The objective of the study is to evaluate the long-term safety and efficacy of a sub-retinal injection of BIIB111 in participants with Choroideremia (CHM) who have been previously treated with BIIB111 and who have exited an antecedent study; these treated participants will be compared with untreated control participants who have exited the STAR (NCT03496012) study and BIIB112 in participants with X-linked retinitis pigmentosa (XLRP) who have been previously treated with BIIB112 and who have exited an antecedent study.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 3
Sex:Male
Key Eligibility Criteria
Disqualifiers:Others
330 Participants Needed
OpCT-001 for Retinitis Pigmentosa
Miami, Florida
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
Key Eligibility Criteria
Disqualifiers:Ocular Inflammation, Glaucoma, Diabetic Retinopathy, Monocular Vision, Others
54 Participants Needed
Gene Therapy for Leber Congenital Amaurosis
Miami, Florida
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+
Key Eligibility Criteria
Disqualifiers:Other Mutations, Infections, Steroid Response, Others
Must Not Be Taking:Corticosteroids
34 Participants Needed
Ultevursen for Retinitis Pigmentosa
Miami, Florida
The purpose of this Phase 2b study is to evaluate the safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. This is a multicenter Double-masked, Randomized, Sham-controlled study which will enroll 81 subjects.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:8+
Key Eligibility Criteria
Disqualifiers:Non-exon 13 Mutations, Other Retinal Diseases, Unstable CME, Others
81 Participants Needed
Electro-acupuncture + TES for Retinitis Pigmentosa
Fort Lauderdale, Florida
This trial is testing a treatment called transcorneal electrical stimulation for people with retinitis pigmentosa, a condition that leads to gradual vision loss. The treatment uses small electrical currents applied to the eye to improve blood flow and stimulate retinal cells, which may help slow down vision loss. Transcorneal electrical stimulation has shown several improvements in visual function in previous studies and is currently offered by several companies.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Key Eligibility Criteria
Disqualifiers:Severe Vision Loss, Dementia, Pregnancy, Others
21 Participants Needed
Stem Cell Therapy for Retinal and Optic Nerve Disorders
Coral Springs, Florida
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
Key Eligibility Criteria
Disqualifiers:Inadequate Exam, No Follow-up, No Consent, Others
500 Participants Needed
Gene Therapy for Retinitis Pigmentosa
Gainesville, Florida
This trial tests a gene therapy for people with X-linked retinitis pigmentosa. It uses a harmless virus to deliver a healthy gene to eye cells, aiming to improve their function and slow down vision loss. Gene therapy targeting the RPE65 gene has shown promise in treating inherited retinal dystrophies, including retinitis pigmentosa.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:3+
97 Participants Needed
4D-125 for Retinitis Pigmentosa
Gainesville, Florida
This trial tests a new eye injection treatment for males with a genetic eye disease. The treatment replaces a faulty gene with a healthy one to help stop or slow down the disease. Gene therapy has been extensively studied and shows promise for treating genetic eye diseases by delivering functional genes to replace faulty ones.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:12+
Sex:Male
Key Eligibility Criteria
Disqualifiers:AAV Treatment, Eye Conditions, Others
21 Participants Needed
AGTC-501 for Retinitis Pigmentosa
Jacksonville, Florida
This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:8 - 50
Sex:Male
Key Eligibility Criteria
Disqualifiers:Herpes, Immunosuppression, Systemic Infection, Others
Must Not Be Taking:Anticoagulants, Corticosteroids, Immunosuppressives, Antivirals
14 Participants Needed
VP-001 for Retinal Dystrophy
Jacksonville, Florida
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+
Key Eligibility Criteria
Disqualifiers:Uncontrolled Systemic Disease, Other RP, Others
Must Not Be Taking:Anti-VEGF, Corticosteroids, Ozurdex
12 Participants Needed
Gene Therapy for Retinitis Pigmentosa
Jacksonville, Florida
This trial is testing a new treatment for people with a genetic eye disease that causes vision loss. The treatment uses a harmless virus to deliver a healthy gene directly into the eye. This could help improve or stabilize their vision. The study will monitor safety and effectiveness over time. This marks a significant advance in treating genetic eye diseases.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Age:6 - 50
Sex:Male
Key Eligibility Criteria
Disqualifiers:Not Listed
29 Participants Needed
Why Other Patients Applied
"I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."
AG
Paralysis PatientAge: 50
"I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."
ID
Pancreatic Cancer PatientAge: 40
"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
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+
Key Eligibility Criteria
Disqualifiers:Other Study, Non-compliance, Others
165 Participants Needed
Oral Metformin for Stargardt Disease
Bethesda, Maryland
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
Key Eligibility Criteria
Disqualifiers:Pregnancy, Chronic Renal Impairment, Type 1 Diabetes, Others
Must Not Be Taking:Cimetidine, Furosemide, Nifedipine, GLP-1 Agonists
56 Participants Needed
N-acetylcysteine for Retinitis Pigmentosa
Baltimore, Maryland
This trial is testing N-acetylcysteine (NAC) tablets to help people with Retinitis Pigmentosa (RP), a disease that causes vision loss. NAC is an antioxidant that protects eye cells from damage. The goal is to see if NAC can slow down or stop the progression of vision loss in RP patients.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Age-related Macular Degeneration, Diabetic Retinopathy, Retinal Vein Occlusion, Others
Must Be Taking:N-acetylcysteine
30 Participants Needed
RTx-015 for Retinitis Pigmentosa
Bellaire, Texas
A Phase 1, open-label, non-randomized, dose-escalation study, where approximately 18 eligible patients with retinitis pigmentosa or choroideremia will be enrolled sequentially in up to 4 dose cohorts of RTx-015. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-015 in the study eye at Visit 3 (Day 0) and be followed for a total of 5 years.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Gene Therapy, Retinal Implant, Others
Must Not Be Taking:Immunosuppressives, Steroids
18 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:Active Not Recruiting
Trial Phase:Phase 1, 2
Key Eligibility Criteria
Disqualifiers:Gene Therapy, Ocular Surgery, Retinal Detachment, Others
10 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
Key Eligibility Criteria
Disqualifiers:Other Gene Mutations, Ocular Disorders, Pregnancy
82 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+
Key Eligibility Criteria
Disqualifiers:Gene Therapy, Investigational Drug, Eye Surgery, Others
Must Not Be Taking:Retinoid Compounds
31 Participants Needed
Gene Therapy for Retinitis Pigmentosa
New York, New York
This trial tests a modified virus that carries a light-sensitive gene to help people with vision problems. The virus delivers this gene to eye cells, making them respond to light and potentially improving vision.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Key Eligibility Criteria
Disqualifiers:Prior AAV Therapy, Nystagmus
20 Participants Needed
Gene Therapy for Retinitis Pigmentosa
Ann Arbor, Michigan
The purpose of this study is to assess the safety and tolerability of subretinal delivery of Adeno-associated Virus Vector (AAV5 hRKp.RPGR) gene therapy in adults and children with X-linked retinitis pigmentosa.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:5+
Sex:Male
42 Participants Needed
jCell for Retinitis Pigmentosa
Oak Park, Illinois
This study evaluates the safety of a single injection of jCell (famzeretcel) comprising 8.8 million (8.8M) retinal progenitor cells over a six-month study period in a cohort of adult subjects with RP. Additionally, changes in visual function will be evaluated at six months between the active treatment group (8.8M jCell) compared to sham-treated controls.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18 - 60
Key Eligibility Criteria
Disqualifiers:Pregnancy, Breastfeeding, Uncontrolled Hypertension, Others
Must Not Be Taking:Corticosteroids, Hydroxychloroquine, Interferon, Others
60 Participants Needed
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