A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Treated Previously with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) in an Antecedent Study (SOLSTICE)
- Sponsor: NightstaRx, Ltd, a Biogen Company
- Principal Investigator: Kimberly Stepien, MD
- Study Coordinator: Nickie Stangel
Study objective:
Long-term follow up evaluating the safety and efficacy of AAV2-REP1 used in antecedent choroideremia treatment studies.
Study design:
The study will consist of up to 9 visits over a maximum 48-month study period. Subjects will rollover into the current study when they complete the antecedent study. No further gene therapy will be given in this protocol.
Inclusion criteria:
- Have received a sub-retinal injection of AAV2-REP1 for CHM in an antecedent study
Exclusion criteria:
- There are no exclusion criteria for this study
Contact Nickie Stangel with questions: 608-263-8783. For more information about this study: visit clinicaltrials.gov