A Phase I/II Study to Assess the Safety and Tolerability of a Single Subretinal Administration of SPVN06 Gene Therapy in Subjects With Rod-Cone Dystrophy (RCD) Due to a Mutation in the RHO, PDE6A, or PDE6B Gene
SparingVision
Summary
This is a two-step, multicenter, Phase I/II study including an open-label dose-escalation phase (Step 1) and a three-arm, controlled, double-masked, randomized extension phase (Step 2), in subjects with advanced RCD due to a mutation in the RHO, PDE6A, or PDE6B gene.
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: Subjects will be eligible to participate in this study only if all the following criteria apply: 1. Able to give signed informed consent and comply with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. 2. Age ≥18 years at the time of ICF signature. 3. Subjects of either gender previously diagnosed with advanced RCD due to biallelic mutations in the rod cGMP phosphodiesterase 6 beta (PDE6B) or rod cGMP phosphodiesterase alpha (PDE6A) genes, or due to a monoallelic dominant mutation in the rhodopsin (RHO) gene. The genotyping…
Interventions
- DrugSPVN06
AAV-RdCVF-RdCVFL
Locations (6)
- Bascom Palmer Eye Institute/University of MiamiMiami, Florida
- Mass Eye and EarBoston, Massachusetts
- Casey Eye InstitutePortland, Oregon
- UPMC Eye CenterPittsburgh, Pennsylvania
- Retina Foundation of the SouthwestDallas, Texas
- CHNO XV-XX Paris - CIC 1423Paris