Actively Recruiting // Clinical Trials // Inherited Retinal Degeneration // Kimberly Stepien // Sep 30 2020

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

Study objective:

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).

Study design:

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.