Trial Profile
A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)
Status:
Completed
Phase of Trial:
Phase I/II
Latest Information Update: 23 Jan 2024
Price :
$35
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At a glance
- Drugs Cotoretigene toliparvovec (Primary)
- Indications Retinitis pigmentosa
- Focus Adverse reactions; First in man; Proof of concept; Therapeutic Use
- Acronyms XIRIUS
- Sponsors Biogen; Nightstar Therapeutics; NightstaRx
- 01 Mar 2023 Results of a post hoc analysis of the XIRIUS and XOLARIS studies published in the JAMA Ophthalmology
- 14 May 2021 Primary endpoint (Part 2: Percentage of Study Eyes with 7 Decibels (dB) Improvement from Baseline at 5 of the 16 Central Loci of the 10-2 Grid Assessed by Macular Integrity Assessment (MAIA) Microperimetry) has not been met as per results published in Biogen media release.
- 14 May 2021 According to Biogen media release, complete analysis of this XIRIUS study is ongoing, and detailed results will be shared in a future scientific forum.