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Retinitis Pigmentosa clinical trials at University of California Health

18 in progress, 7 open to eligible people

Showing trials for
  • 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

  • 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!”

    open to eligible people ages 18 years and up

    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

  • The Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

    open to eligible people ages 18 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 Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)

    open to eligible people ages 5 years and up

    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 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

  • 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 of PRPF31 Mutation-Associated Retinal Dystrophy

    Sorry, not currently recruiting here

    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

  • Natural History of the Progression of X-Linked Retinitis Pigmentosa

    Sorry, in progress, not accepting new patients

    The objective of the study is to gain a better understanding of disease progression over time in participants with X-linked retinitis pigmentosa (XLRP).

    at UCLA UCSF

  • 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, not yet accepting 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 .

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