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AAV5-RPGR · 1 trial · 1 indication
The primary outcome is defined as any of the below occurring during the 9 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Product (ATIMP), not surgery alone: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more * Severe unresponsive inflammation * Infective endophthalmitis * Ocular malignancy * Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)
| Arm | Type | Description |
|---|---|---|
| Phase 1 (Part 1, Dose Escalation) | EXPERIMENTAL | Participants receive one of three doses of AAV5-RPGR |
| Phase 2 (Part 2; Expansion) | EXPERIMENTAL | Participants receive one of two doses of AAV5-RPGR |
| Name | Type | Description |
|---|---|---|
| AAV5-RPGR | GENETIC | Single, subretinal administration of AAV5-RPGR |
Key inclusion Criteria: * Males aged 5 years or older * Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI) Key exclusion Criteria: • Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months
AAV5-RPGR is an investigational gene therapy being developed for X-linked retinitis pigmentosa, a rare inherited retinal disease. It is designed to address the genetic cause of vision loss in patients with mutations in the RPGR gene. The therapy has been studied in a Phase 1 clinical trial.
AAV5-RPGR is being developed by MeiraGTx Holdings plc, a clinical-stage gene therapy company. The company's ticker symbol is MGTX. MeiraGTx is focused on developing gene therapies for serious diseases, including inherited retinal dystrophies such as X-linked retinitis pigmentosa.
AAV5-RPGR is in Phase 1 clinical development. A single Phase 1 trial has been completed. The trial was a controlled, randomized study that enrolled 49 patients with X-linked retinitis pigmentosa. It was conducted in the United States and the United Kingdom.
AAV5-RPGR has been evaluated in one clinical trial, NCT03252847, titled "Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)." This Phase 1 trial is completed and enrolled 49 male patients aged 5 years and older with X-linked retinitis pigmentosa.
AAV5-RPGR is not approved by the FDA. It is an investigational gene therapy that has completed a Phase 1 clinical trial. Further clinical development would be required before any potential regulatory approval. The trial was conducted in the United States and the United Kingdom.