An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2-.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
The Phase 1 part of the study is a dose escalation of subretinal administration of AAV2/5 vector to assess the safety of this vector in participants with XLRP caused by mutations in RPGR. The Phase 2 part of the study is a cohort expansion of subretinal administration of AAV2/5 vector to assess the safety and efficacy of this vector in participants with XLRP caused by mutations in RPGR.
NCT03252847 — X-Linked Retinitis Pigmentosa
Status: Completed
http://inclinicaltrials.com/x-linked-retinitis-pigmentosa/NCT03252847/
Retinitis Pigmentosa Clinical Study: Examining the Clinical Trial Journey of Retinitis Pigmentosa Patients
Typically, clinical research participation favors a specific demographic group, and little research exists on how trial attributes affect participation. As such, this study seeks to analyze data from different demographic groups and check for recurring trends that could provide valuable insights for future retinitis pigmentosa patients.
NCT05849987 — Retinitis Pigmentosa
Status: Not yet recruiting
http://inclinicaltrials.com/retinitis-pigmentosa/NCT05849987/
Phase 3 Study to Evaluate the Safety and Efficacy of AAV5-hRKp.RPGR for the Treatment of Japanese X-linked Retinitis Pigmentosa Associated With Pathogenic Variants in Retinitis Pigmentosa GTPase Regulator (RPGR)
The purpose of the study is to assess the safety and tolerability of bilateral subretinal delivery of adeno-associated virus vector with a serotype 5 capsid human rhodopsin kinase promoter. retinitis pigmentosa guanosine triphosphatase regulator (AAV5-hRKp.RPGR).
NCT05926583 — X-Linked Retinitis Pigmentosa
Status: Recruiting
http://inclinicaltrials.com/x-linked-retinitis-pigmentosa/NCT05926583/
Prospective Observational Study to Estimate the Genotype and Phenotype of Japanese Patients With X-linked Retinitis Pigmentosa Associated With Pathogenic Variants in Retinitis Pigmentosa GTPase Regulator (RPGR)
The purpose of the study is to identify a cohort of Japanese participants with X-linked retinitis pigmentosa (XLRP) associated with pathogenic variants in the retinitis pigmentosa GTPase regulator (RPGR) gene and to investigate their associated phenotype.
NCT04868916 — X-Linked Retinitis Pigmentosa
Status: Completed
http://inclinicaltrials.com/x-linked-retinitis-pigmentosa/NCT04868916/
Phase 1, Open-Label, Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-015 in Patients With Retinitis Pigmentosa
A Phase 1, open-label, non-randomized, dose-escalation study, where approximately 9 eligible patients with retinitis pigmentosa will be enrolled sequentially in up to 3 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 12 months.
NCT06460844 — Retinitis Pigmentosa
Status: Not yet recruiting
http://inclinicaltrials.com/retinitis-pigmentosa/NCT06460844/
A Phase 3, Multi-Center, Randomized Study to Assess The Efficacy, Safety and Tolerability of Subretinal OCU400 Gene Therapy for the Treatment of Retinitis Pigmentosa
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.
NCT06388200 — Retinitis Pigmentosa
Status: Recruiting
http://inclinicaltrials.com/retinitis-pigmentosa/NCT06388200/
Observational, Non-Interventional Study to Determine the Operational Feasibility and Measurement Properties of Endpoints in Patients With Retinitis Pigmentosa
RayTx established The Vision Research and Assessment Institute (VRAI) with the express purpose of serving as a testing facility for efficacy endpoints for patients with Low Vision. The mission of the VRAI is to enable the highest quality, standardized efficacy testing of patients with visual impairment to be performed by expert clinicians in an optimized environment. The proposed assessments or testing modalities to be evaluated in this non-interventional study are published in some capacity, and some of these publications already include patients with severe-to-profound vision loss from retinitis pigmentosa and other inherited retinal diseases.
NCT06375239 — Retinitis Pigmentosa
Status: Recruiting
http://inclinicaltrials.com/retinitis-pigmentosa/NCT06375239/
Clinical and Genetic Findings in Patients With PRPF31-associated Retinitis Pigmentosa
Retrospective chart review study to elucidate the genotype and phenotype of patients with PRPF31-associated retinitis pigmentosa and asymptomatic carriers of the respective variant(s)
NCT06368375 — Retinitis Pigmentosa
Status: Completed
http://inclinicaltrials.com/retinitis-pigmentosa/NCT06368375/
Exploration of New Sensitive Clinical Readouts and Biomarkers That Can be Used as Clinical Endpoints Tailored to Monitor Treatment Effects in PDE6A-, PDE6B- and RHO-linked Retinitis Pigmentosa: a Non-interventional Trial
The aim of the study is to apply a novel clinical investigation protocol in patients with Phosphodiesterase 6A (PDE6A), PDE6B and Rhodopsin (RHO)-based retinitis pigmentosa. This novel, multimodal clinical examination protocol describes and correlates structural, functional and metabolic aspects during natural disease development. Test-retest variability of new measurements as well as correlations of the structural, functional, and metabolic changes will be defined to be able to define well-suited readouts for safety and efficacy of future treatment developments before they reach the clinical phase.
NCT06323772 — Retinitis Pigmentosa
Status: Recruiting
http://inclinicaltrials.com/retinitis-pigmentosa/NCT06323772/
Biomarkers for Prognosis in Different Forms of Retinitis Pigmentosa
The objective of this study is to discover biomarkers that demonstrate a correlation between the severity of retinitis pigmentosa (RP) and the thickness of the retinal pigment epithelium (RPE). These biomarkers will serve as prognostic indicators for various kinds of retinitis pigmentosa. The objective of this study is to find biomarkers that establish a correlation between the severity of retinitis pigmentosa and the thickness of the retinal pigment epithelium (RPE), which can serve as a prognostic indicator for Retinitis Pigmentosa.
NCT06306690 — Retinitis Pigmentosa
Status: Recruiting
http://inclinicaltrials.com/retinitis-pigmentosa/NCT06306690/