An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Efficacy of LX103 in Patients With X-Linked Retinoschisis (XLRS)
Status: Recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Not Applicable
SUMMARY
The goal of this study is to evaluate the safety and efficacy of LX103 treatment of X-linked retinoschisis. This study will enroll subjects aged ≥ 6 years old to receive a single unilateral intravitreal (IVT) injection of LX103 to evaluate its safety and efficacy.
Eligibility
Participation Requirements
Sex: Male
Minimum Age: 6
Healthy Volunteers: f
View:
• Willing to sign the informed consent, and willing to attend follow-up visits.
• Male individual at least 6 years of age with a diagnosis of XLRS and documented mutations in the RS1 gene.
• The study eye must have a best-corrected E-ETDRS visual acuity letter score of less than or equal to 63.
Locations
Other Locations
China
Shanghai General Hospital, Shanghai Jiao Tong University
RECRUITING
Shanghai
Time Frame
Start Date: 2022-12-13
Estimated Completion Date: 2028-08
Participants
Target number of participants: 12
Treatments
Experimental: LX103 Injection
Potential doses:~5E10 vg, 0.1 mL/eye/dose (low dose)~1E11 vg, 0.05 mL/eye/dose (high dose)
Related Therapeutic Areas
Sponsors
Leads: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Collaborators: Innostellar Biotherapeutics