Clinical Research Directory

Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)

This study is an early-stage clinical trial (Phase 1b/2a) testing a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare genetic eye disease that leads to severe vision loss. The treatment is delivered as a one-time injection (300 µL) into the retina (subretinal space) of the worse-seeing eye, using a method similar to approved gene therapies like Luxturna. The study is designed to evaluate safety and effectiveness at two dose levels (1E11 and 3E11 viral genomes per eye) in small groups of 5 participants. Each group begins cautiously with 2 adults (age ≥18), treated at least one month apart, followed by FDA review before allowing adolescents (ages 12-17) to participate. An independent monitoring committee (IDMC) oversees safety throughout. After 3 adolescents are treated and followed for 3 months, the committee reviews all data to decide whether to move to a higher dose. However, if the lower dose (1E11 vg/eye) shows strong effectiveness in the first group, the study may expand by treating more adolescents at that same dose instead of increasing it further.

Psychotherapy Group for Parents of Children With LCA

The objectives of this study are to evaluate the effect of a group psychotherapy intervention for parents of children/adolescents with LCA on indicators of emotional health, parental stress, acceptance, cognitive defusion, and mindfulness. The study design is a repeated measures design with pre-, post-, and 3-month follow-up assessments. Forty parents of children and adolescents with LCA will be included in the sample. Twenty will participate in the psychotherapy groups, divided into 2 groups of 10 people, and 20 will be in the control group. To assess the indicators mentioned in the objective, the following questionnaires will be applied: Self-Reporting Questionnaire, Parental Stress Scale, Acceptance and Action Questionnaire, Cognitive Fusion Questionnaire, and Freiburg Mindfulness Inventory. A subsequent data analysis will be conducted to evaluate and correlate these indicators before, after, and 3 months post-intervention.