A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa (XIRIUS)
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)
Researchers in PA, OR, and FL are recruiting male X-Linked Retinitis Pigmentosa patients for an investigation of a gene therapy designed to impart a functional retinitis pigmentosa GTPase regulator gene (RPGR) to correct the disease. The treatment would require a single intravitreal (into the retina) injection of the drug, a genetically modified and harmless virus, at 1 of 6 dosage levels. The patient will be followed for ~ 2 years to assess for adverse safety events.
- Be 10 years of age or older and male
- Have documentation of a mutation in the RPGR gene
- Not have participated in a gene therapy trial previously or a clinical trial with an investigational drug in the past 12 weeks
THE STUDY INVOLVES:
- Prescreening tests to confirm eligibility of the patient to participate.
- Intravitreal injection of the gene therapy at one of several dose levels.
- Follow up for ~ 2 years to screen for adverse safety events
LOCATIONS AND CONTACTS:
The study sites are not yet disclosed. Contact the study’s contact to qualify.
Nightstar Therapeutics | +44 207 611 2077 (international) | [email protected]
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