Retinitis Pigmentosa clinical trials at University of California Health
17 in progress, 6 open to eligible people
Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
open to eligible people ages 3 years and up
A clinical trial of AAV5-RPGR vector for patients with X-linked retinitis pigmentosa (XLRP)
at UCSD
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
open to eligible people ages 3 years and up
A clinical trial of AAV5-RPGR vector for patients with X-linked retinitis pigmentosa (XLRP)
at UCSD
The Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
open to eligible people ages 6 years and up
To assess the safety of unilateral subretinal administration of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patients with LCA due to mutation(s) in CEP290 gene (LCA10).
at UCSD
Natural History of PRPF31 Mutation-Associated Retinal Dystrophy
open to eligible people ages 10 years and up
The purpose of this study is to characterize the natural history through temporal systemic evaluation of subjects identified with PRPF31 mutation-associated retinal dystrophy, also called retinitis pigmentosa type 11, or RP11. Assessments will be completed to measure and evaluate structural and functional visual changes including those impacting patient quality of life associated with this inherited retinal condition and observing how these changes evolve over time.
at UCSF
Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F
open to eligible people ages 8 years and up
The overall goal of this project, co-funded by the Foundation Fighting Blindness and the USHER 1F Collaborative is to characterize the natural history of disease progression in patients with PCDH15 mutations in order to accelerate the development of outcome measures for clinical trials.
at UCSF
Retinal Imaging in Patients With Inherited Retinal Degenerations
open to eligible people ages 13 years and up
The purpose of this study is to determine whether the structure and function of the human retina can be studied with high resolution in patients with inherited retinal degenerations using the Adaptive Optics Scanning Laser Ophthalmoscope (AOSLO).
at UCSF
Advancing Understanding of Transportation Options
Sorry, in progress, not accepting new patients
This Stage II randomized, controlled, longitudinal trial seeks to assess the acceptability, feasibility, and effects of a driving decision aid use among geriatric patients and providers. This multi-site trial will (1) test the driving decision aid (DDA) in improving decision making and quality (knowledge, decision conflict, values concordance and behavior intent); and (2) determine its effects on specific subpopulations of older drivers (stratified for cognitive function, decisional capacity, and attitudinally readiness for a mobility transition). The overarching hypotheses are that the DDA will help older adults make high-quality decisions, which will mitigate the negative psychosocial impacts of driving reduction, and that optimal DDA use will target certain populations and settings.
at UCSD
Clinical Trial of Stem Cell Use in Vision Loss
“We hope to learn more about the safety and feasibility of using your own (autologous) stem cells extracted from your bone marrow.”
Sorry, accepting new patients by invitation only
This pilot study is to determine whether it would be safe and feasible to inject CD34+ stem cells from bone marrow into the eye as treatment for patients who are irreversibly blind from various retinal conditions.
at UC Davis
Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa
Sorry, accepting new patients by invitation only
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.
at UCLA
Oral N-acetylcysteine for Retinitis Pigmentosa
Sorry, not yet accepting patients
Retinitis pigmentosa (RP) is an inherited retinal degeneration caused by one of several mistakes in the genetic code. Such mistakes are called mutations. The mutations cause degeneration of rod photoreceptors which are responsible for vision in dim illumination resulting in night blindness. After rod photoreceptors are eliminated, gradual degeneration of cone photoreceptors occurs resulting in gradual constriction of side vision that eventually causes tunnel vision. Oxidative stress contributes to cone degeneration. N-acetylcysteine (NAC) reduces oxidative stress and in animal models of RP it slowed cone degeneration. In a phase I clinical trial in patients with RP, NAC taken by month for 6 months caused some small improvements in two different vision tests suggesting that long-term administration of NAC might slow cone degeneration in RP. NAC Attack is a clinical trial being conducted at many institutions in the US, Canada, Mexico, and Europe designed to determine if taking NAC for several years provides benefit in patients with RP.
at UC Davis UCSF
RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa
Sorry, in progress, not accepting new patients
Currently enrolling a total of 12 patients for Phase 2a of the study: 6 patients must have VA of no-better-than hand motion in the study eye, and 6 patients must have VA in the study eye to range from no-worse-than count fingers to 20/200 vision.
at UCSF
Stem Cell Therapy Study for Vision Loss from Retinitis Pigmentosa
“Volunteer for research and contribute to discoveries that may improve health care for you, your family, and your community!”
Sorry, in progress, not accepting new patients
In this Phase 1 open-labeled prospective study, one eye of each participant with vision loss from retinitis pigmentosa will be administered intravitreal injection of autologous CD34+ stem cells harvested from bone marrow. Each participant will be examined serially for 6 months after study injection to determine safety and feasibility of this intervention.
at UC Davis
Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)
Sorry, in progress, not accepting new patients
The purpose of this study is to evaluate the efficacy 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.
at UCSD UCSF
Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)
Sorry, in progress, not accepting new patients
The rod-cone dystrophies (often referred to as retinitis pigmentosa (RP)) are a clinically and genetically heterogeneous group of disorders in which there is progressive loss of rod and later cone photoreceptor function leading to severe visual impairment. RP usually occurs as an isolated retinal disorder, but it may also be seen in association with systemic abnormalities.
at UCSD
Rate of Progression in EYS Related Retinal Degeneration
Sorry, in progress, not accepting new patients
The overall goal of this project funded by the Foundation Fighting Blindness is to characterize the natural history of disease progression in patients with EYS mutations in order to accelerate the development of outcome measures for clinical trials.
at UCSF
Rate of Progression in USH2A-related Retinal Degeneration
Sorry, in progress, not accepting new patients
The overall goal of this project funded by the Foundation Fighting Blindness is to characterize the natural history of disease progression in patients with USH2A related retinal degeneration associated with congenital hearing loss (Usher syndrome type 2a) or non-syndromic retinitis pigmentosa (RP39).
at UCSF
Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants
Sorry, in progress, not accepting new patients
This is an international, multicenter study with two components: Registry - A standardized genetic screening and a prospective, standardized, cross-sectional clinical data collection - Enrollment is open to all genes on the RD Rare Gene List Natural History Study - A prospective, standardized, longitudinal Natural History Study - Enrollment opens gene-by-gene, based on funding and within-gene Registry enrollment The study objectives are as follows. Registry Objectives 1. Genotype Characterization 2. Cross-Sectional Phenotype Characterization (within gene) 3. Establish a Link to My Retina Tracker Registry (MRTR) 4. Ancillary Exploratory Studies - Pooling of Genes Natural History Study Objectives 1. Natural History (within gene) 2. Structure-Function Relationship (within gene) 3. Risk Factors for Progression (within gene) 4. Ancillary Exploratory Studies - Pooling of Genes
at UCSD UCSF
Our lead scientists for Retinitis Pigmentosa research studies include Susanna Park, MD PhD Jacque Duncan, MD.
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