Clinical trials: X-Linked Retinitis Pigmentosa (XLRP)
X-Linked Retinitis Pigmentosa
Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
Phase 1 of the study is a dose escalation of the subretinal administration of AAV5-hRKp.RPGR vector to assess the safety of this vector in participants with XLRP caused by mutations in RPGR.
- Trial name
- NCT03252847
- Testing
- AAV5-RPGR
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 49 people
- Sponsor
- MeiraGTx UK II Ltd
X-Linked Retinitis Pigmentosa
A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
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…
- Trial name
- NCT04850118
- Testing
- rAAV2tYF-GRK1-hRPGRco, Control
- Where
- 25 sites in United States, Australia and 2 more
- Volunteers
- 85 people
- Sponsor
- Beacon Therapeutics
X-Linked Retinitis Pigmentosa (XLRP)
A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa
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…
- Trial name
- Landscape (NCT07174726)
- Testing
- Adeno-associated virus vector expressing a human RPGR gene
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Beacon Therapeutics
X-Linked Retinitis Pigmentosa
A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112
The objective of the study is to evaluate the safety, tolerability and efficacy of a single sub-retinal injection of BIIB112 in participants with X-linked retinitis pigmentosa (XLRP).
- Trial name
- XIRIUS (NCT03116113)
- Testing
- BIIB112
- Where
- 8 sites in United States, United Kingdom
- Volunteers
- 50 people
- Sponsor
- Biogen
X-Linked Retinitis Pigmentosa
A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)
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.
- Trial name
- NCT06646289
- Testing
- AAV5-hRKp.RPGR, No intervention (Follow-Up assessment)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 24 people
- Sponsor
- MeiraGTx Ocular UK Ltd
X-Linked Retinitis Pigmentosa
Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)
The purpose of this study is to evaluate subjects with X-linked retinitis pigmentosa caused by RPGR-ORF15 mutations in a clinical setting to fully characterize their condition, measure testing variability, and estimate rates of…
- Trial name
- NCT03314207
- Where
- 3 sites in United States
- Volunteers
- 14 people
- Sponsor
- Beacon Therapeutics
X-Linked Retinitis Pigmentosa (XLRP)
Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa
The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene…
- Trial name
- NCT06492850
- Testing
- FT-002
- Where
- China
- Volunteers
- 32 people
- Sponsor
- Frontera Therapeutics
X-Linked Retinitis Pigmentosa
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
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.
- Trial name
- NCT06275620
- Testing
- AGTC-501 (high dose and standard corticosteroid regimen), AGTC-501 (low dose and standard corticosteroid regimen), AGTC-501 (high dose and modified corticosteroid regimen)
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Beacon Therapeutics
X-Linked Retinitis Pigmentosa
Natural History of the Progression of X-Linked Retinitis Pigmentosa
The objective of the study is to gain a better understanding of disease progression over time in participants with X-linked retinitis pigmentosa (XLRP).
- Trial name
- XOLARIS (NCT04926129)
- Testing
- Other: Assessments
- Where
- 22 sites in United States, Canada and 5 more
- Volunteers
- 201 people
- Sponsor
- NightstaRx Ltd, a Biogen Company
X-Linked Retinitis Pigmentosa
A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)
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.
- Trial name
- NCT06333249
- Testing
- rAAV2tYF-GRK1-RPGR
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- Beacon Therapeutics
RP2-associated Retinitis Pigmentosa, X-Linked Retinitis Pigmentosa (XLRP)
InsightRP2 Registry
InsightRP2 is a secure online patient registry specific to RP2-associated retinitis pigmentosa (RP). It is our goal to further the scientific understanding of this rare disease and to support research in to a gene therapy for…
- Conditions
- RP2-associated Retinitis Pigmentosa, X-Linked Retinitis Pigmentosa (XLRP), Retinitis Pigmentosa 2
- Trial name
- NCT06982417
- Where
- Germany
- Volunteers
- 200 people
- Sponsor
- University of Göttingen
X-Linked Retinitis Pigmentosa
Long-term Follow-up Gene Therapy Study for RPGR- XLRP
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.
- Trial name
- NCT04312672
- Testing
- AAV5-hRKp.RPGR
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 42 people
- Sponsor
- MeiraGTx Ocular UK Ltd
X-Linked Retinitis Pigmentosa
4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
This is a Phase 1/2 multicenter study with two parallel parts: an observational natural history cohort and an open-label, prospective interventional trial in males with non-syndromic X-linked retinitis pigmentosa (XLRP) due to…
- Trial name
- NCT04517149
- Testing
- 4D-125 IVT Injection, Observational
- Where
- 8 sites in United States
- Volunteers
- 21 people
- Sponsor
- 4D Molecular Therapeutics
X-Linked Retinitis Pigmentosa
Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
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.
- Trial name
- HORIZON (NCT03316560)
- Testing
- rAAV2tYF-GRK1-RPGR
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Beacon Therapeutics