A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study (Solstice)
Sponsor: NightstaRx, Ltd
Principal Investigator: Kimberly Stepien, MD
Study Coordinator: Nickie Stangel
This study will evaluate the long-term safety and efficacy of AAV2-REP1 in subjects with choroideremia (CHM), and AAV8-RPGR in subjects with X-linked retinitis pigmentosa (XLRP).
This is a multi-center, observational, follow-up study for subjects who have previously received a sub-retinal injection of AAV2-REP1 for the treatment of CHM or a sub-retinal injection of AAV8-RPGR for the treatment of XLRP.
Diagnosis and Key Eligibility Criteria:
Have received AAV2-REP1 for CHM or AV8-RPGR for XLRP and have exited an antecedent study.
Duration of study: Approximately 4 years.
For more information, contact Nickie Stangel 608-263-8783.