A Dose Escalation, Phase 1/2 Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)
Phase of Trial: Phase I/II
Latest Information Update: 03 Apr 2018
At a glance
- Drugs AAV RPGR Gene Therapy NightstaRx (Primary)
- Indications Retinitis pigmentosa
- Focus Adverse reactions; First in man
- Acronyms XIRIUS
- Sponsors Nightstar Therapeutics; NightstaRx
- 03 Apr 2018 According to a Nightstar Therapeutics media release, the company expects the initial data on safety and tolerability from the dose escalation cohorts of this trial to be available towards the end of 2018; this data will determine the dose for study in an expansion cohort anticipated to include up to 30 patients.
- 22 Mar 2017 New trial record
- 19 Mar 2017 According to a NightstaRx media release, the company has commenced enrolling and dosing subjects in this trial.