Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa

Launched by NIGHTSTARX LTD, A BIOGEN COMPANY · Jun 29, 2018

Keywords

ClinConnect Summary

SOLSTICE is a long-term follow-up study looking at safety and how well two gene therapies may work for inherited eye diseases: BIIB111 for Choroideremia (CHM) and BIIB112 for X-linked Retinitis Pigmentosa (XLRP). The study will enroll adult men who have already received BIIB111 or BIIB112 in earlier trials and completed those studies, plus a comparison group of untreated participants who also left prior studies. This isn’t a new randomized trial; it’s designed to track safety and ongoing effects over several years. There are many study sites around the world, and the plan is to enroll about 330 participants.

If you join, you can expect up to five years of scheduled eye visits and tests. These visits will include checks of eye pressure, eye surface health, the lens, photos of the back of the eye, and advanced eye scans to look at retinal structure. Vision tests will measure how well you see at various distances and how your daily vision feels, with some tests performed after dilation of the pupils. For those in the XLRP group, additional vision tests are included. The main focus is safety (any adverse events) and how vision and retinal health may change over time, with several secondary measures looking at small vision changes and quality of life. The trial is non-randomized, and some outcome assessments may be masked to treatment to reduce bias. The study is ongoing through 2026 at centers in North America, Europe, Brazil, and other locations.

Gender

MALE

Eligibility criteria

• Key Inclusion Criteria:
• • CHM Participants
• • a. Have participated in and exited from an interventional study that investigated the safety and efficacy of a sub-retinal injection of BIIB111 for CHM.
• • XLRP Participants
• • a. Have received a sub-retinal injection of BIIB112 for XLRP and have exited an antecedent study.
• Key Exclusion Criteria:
• • Participants are not eligible for study participation if they meet the following exclusion criterion.
• • a. In the opinion of the Investigator and/or the Sponsor, it is not in the participant's best interest to participate in the study.
• • NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.