Evaluating LX101 for inherited retinal dystrophy in the other eye
A Clinical Trial to Evaluate the Safety and Efficacy of Subretinal Re-Administration of LX101 to the Contralateral Eye in Subjects With Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy
This study is testing if a gene therapy called LX101 is safe and effective when given to the other eye of people with inherited retinal dystrophy who have already received the treatment in one eye.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 9 (estimated) |
| Ages | 6 Years and up |
| Sex | All |
| Sponsor | Innostellar Biotherapeutics Co.,Ltd Industry-sponsored |
| Locations | 1 site (Shanghai, Shanghai) |
| Trial ID | NCT06212297 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to assess the safety of administering LX101, a gene therapy, in the previously untreated eye of subjects who have already received the treatment in one eye. Up to nine participants with inherited retinal dystrophy, specifically those with RPE65 mutations, will be involved in this interventional study. The trial will focus on the sequential subretinal administration of LX101 to evaluate its effects and safety profile. Participants must meet specific eligibility criteria, including prior treatment with LX101 and being at least six years old.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals aged six years and older who have previously received unilateral LX101 treatment.
Not a fit: Patients who have undergone other gene therapies or have active ocular infections may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve vision in patients with inherited retinal dystrophy by providing a new therapeutic option.
How similar studies have performed: Other studies involving gene therapy for inherited retinal dystrophy have shown promising results, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: Prior subretinal administration of LX101 (unilateral) ≥ 6 years old Signed written informed consent Exclusion Criteria: Prior gene therapy except LX101 Active intraocular or periocular infections Lacking of sufficient surviving retinal cells Prior ocular surgery within six months Retinoid like compounds or precursors were taken within three months Complicating systemic diseases Clinically significant abnormal baseline laboratory values Using of any retinal toxic compounds
Where this trial is running
Shanghai, Shanghai
- Shanghai General Hospital — Shanghai, Shanghai, China (Recruiting)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.