NCT03584165

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

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

• Status: Enrolling By Invitation
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 330 (estimated)
• Sponsor: NightstaRx Ltd, a Biogen Company · Academic / Other
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The objective of the study is to evaluate the long-term safety and efficacy of a sub-retinal injection of BIIB111 in participants with Choroideremia (CHM) who have been previously treated with BIIB111 and who have exited an antecedent study; these treated participants will be compared with untreated control participants who have exited the STAR (NCT03496012) study and BIIB112 in participants with X-linked retinitis pigmentosa (XLRP) who have been previously treated with BIIB112 and who have exited an antecedent study.

Detailed description

This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.

Conditions

• Choroideremia
• X-Linked Retinitis Pigmentosa

Interventions

Type	Name	Description
GENETIC	BIIB111	Administered as specified in the treatment arm.
GENETIC	BIIB112	Administered as specified in the treatment arm.

Timeline

• Start date: 2018-06-04
• Primary completion: 2026-06-04
• Completion: 2026-06-04
• First posted: 2018-07-12
• Last updated: 2025-06-12

Locations

25 sites across 9 countries: United States, Brazil, Canada, Denmark, Finland, France, Germany, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study