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Retinitis Pigmentosa clinical trials

View clinical trials related to Retinitis Pigmentosa.

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NCT ID: NCT04356716 Active, not recruiting - Clinical trials for Age-related Macular Degeneration

Sildenafil for Treatment of Choroidal Ischemia

Start date: November 11, 2014
Phase: Phase 2
Study type: Interventional

The hypothesis of this study is to determine if there is a benefit afforded by the use of systemic Sildenafil to patients with choroidal and retinal degenerations and dystrophies, such as vitelliform degeneration, dry and reticular age-related macular degeneration (AMD) as well as patients with hereditary and acquired retinal dystrophies such as retinitis pigmentosa and central serous retinopathy.

NCT ID: NCT04355689 Active, not recruiting - Usher Syndromes Clinical Trials

Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome

SLO RP
Start date: September 3, 2020
Phase: Phase 1/Phase 2
Study type: Interventional

This study will examine the safety and efficacy of NPI-001 Tablets as compared to placebo for 24 months in subjects with vision loss due to RP associated with Usher syndrome.

NCT ID: NCT04312672 Active, not recruiting - Clinical trials for X-Linked Retinitis Pigmentosa

Long-term Follow-up Gene Therapy Study for RPGR- XLRP

Start date: July 31, 2017
Phase:
Study type: Observational

This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.

NCT ID: NCT04285398 Active, not recruiting - Clinical trials for Retinitis Pigmentosa

Prospective Natural History Study of Retinitis Pigmentosa

PHENOROD2
Start date: February 12, 2020
Phase: N/A
Study type: Interventional

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.

NCT ID: NCT04127006 Active, not recruiting - Clinical trials for Retinitis Pigmentosa

Rate of Progression in EYS Related Retinal Degeneration

Pro-EYS
Start date: February 25, 2020
Phase:
Study type: Observational

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.

NCT ID: NCT04123626 Active, not recruiting - Eye Diseases Clinical Trials

A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene

AURORA
Start date: October 7, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

This study evaluates the safety, tolerability and efficacy of QR-1123 injection in the eye (intravitreal; IVT) injections (one eye/unilateral) in subjects receiving a single dose or repeat doses. Single injections will be assessed in an open label way, and repeat injections will be assessed in a double-masked, randomized, sham-controlled fashion.

NCT ID: NCT03999021 Active, not recruiting - Clinical trials for Retinitis Pigmentosa

FIGHT-RP 1 Extension Study

Start date: June 24, 2019
Phase: Phase 1
Study type: Interventional

Retinitis Pigmentosa (RP) is a devastating eye disease and at present there are no known treatment options that can alter the rate of vision loss and eventual blindness. In a series of studies in animal models, the effects of exposing cones in the periphery of the retina to a large excess of oxygen results in progressive oxidative damage to cone photoreceptors and cone cell death. Cone cell death gradually spreads from the periphery of the retina toward its center, narrowing the visual field and eventually resulting in tunnel vision. Compared to control patients, those with RP showed significant reduction in the reduced to oxidized glutathione ratio (GSH/GSSG) in aqueous humor and a significant increase in protein carbonyl content. This demonstration of oxidative stress and oxidative damage in the eyes of patients with RP, suggests that oxidative damage-induced cone cell death in animal models of RP may translate to humans with RP and support the hypotheses that (1) potent antioxidants will promote cone survival and function in patients with RP and (2) aqueous GSH/GSSG ratio and carbonyl content on proteins provide useful biomarkers of disease activity in this patient population. Orally administered N-acetylcysteine (NAC) has been found to be a particularly effective antioxidant that promotes prolonged cone survival and maintenance of cone function in a mouse model of RP. Since oral and/or topical administration of NAC is feasible for long-term treatment in humans, and NAC has a good safety profile, there is good rationale to test the effect of NAC in patients with RP. Oxidative damage has been implicated in several diseases including cystic fibrosis, chronic obstructive pulmonary disease (COPD), and Idiopathic Pulmonary Fibrosis. The effect of oral NAC has been tested in these indications in several clinical trials providing extensive safety data. In COPD, NAC 600mg bid improves airway function and reduces the frequency of acute exacerbations. Doses of up to 1800mg/day have been well-tolerated in the treatment of Idiopathic Pulmonary Fibrosis. Paracetamol (acetaminophen) toxicity is treated with a loading dose of 140 mg/kg NAC followed by 70 mg/kg every 4 hours for 17 doses. Normal volunteers tolerated a dose of 11.2 grams NAC/day for three months without any serious undesirable effects and in another study a dose of 500mg/kg/day was tolerated. The most frequent adverse events associated with the oral administration of NAC are gastrointestinal in nature and include vomiting, diarrhea, stomatitis, abdominal pain and nausea (incidence rate >1/1000 to <1/100). Hypersensitivity reactions including anaphylactic shock and anaphylactic/anaphylactoid reaction (incidence rate <1/10,000), dyspnea, bronchospasm (incidence rate >1/10,000 to <1/1000), angioedema, tachycardia, urticaria, rash and pruritus (incidence rate >1/1000 to <1/100) have been reported less frequently. Finally, reports of headache, tinnitus, pyrexia, blood pressure decreased (incidence rate >1/1000 to <1/100), face edema and hemorrhage have also been collected with oral NAC. In the FIGHT-RP 1 Study, the investigators used escalating doses of NAC effervescent tablets (from 600 mg in Cohort 1 to 1800 mg in Cohort 3). The maximum tolerated dose was 1800 mg twice a day which will be continued in this study.

NCT ID: NCT03975543 Active, not recruiting - Clinical trials for Retinitis Pigmentosa (RP)

Retrospective Natural History Study of Retinitis Pigmentosa

PHENOROD1
Start date: October 1, 2018
Phase:
Study type: Observational

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.

NCT ID: NCT03963154 Active, not recruiting - Clinical trials for Retinitis Pigmentosa

Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation

Start date: August 19, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

Phase I/II open-label, safety, tolerability and preliminary efficacy study of implantation into one eye of hESC-derived RPE (Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE)) in patients with retinitis pigmentosa due to monogenic mutation. Study non randomized single group assignment consisting in 2 sequential cohorts of patients: - First cohort of 2 patients with very advanced loss of visual acuity (legally blind) - Second cohort of 10 patients with less advanced loss of visual acuity:

NCT ID: NCT03374657 Active, not recruiting - Clinical trials for Retinitis Pigmentosa

A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa

Start date: August 22, 2018
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this first-in-human study is to explore the maximum tolerated dose (MTD) of CPK850 as determined by the single ascending dose ranging portion of the study. This study will also evaluate the safety and potential efficacy of CPK850 on improving visual function in patients with decreased visual function from RLBP1 retinitis pigmentosa due to biallelic mutations in the RLBP1 gene.