Summary

Eligibility
for males ages 10 years and up (full criteria)
Location
at UCLA
Dates
study started
estimated completion

Description

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.

Official Title

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

Details

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

Keywords

Choroideremia X-Linked Retinitis Pigmentosa NightstaRx Biogen NSR-REP1 CHM Gene Therapy AAV REP1 AAV8 RPGR Retinitis Retinitis Pigmentosa BIIB111 BIIB112 Untreated

Eligibility

You can join if…

Open to males ages 10 years and up

CHM Participants

  1. 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

  1. Have received a sub-retinal injection of BIIB112 for XLRP and have exited an antecedent study.

You CAN'T join if...

Participants are not eligible for study participation if they meet the following exclusion criterion.

  1. 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.

Locations

  • Research Site
    Los Angeles California 90095-7065 United States
  • Research Site
    Portland Oregon 97239 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
NightstaRx Ltd, a Biogen Company
ID
NCT03584165
Phase
Phase 3 Research Study
Study Type
Interventional
Participants
Expecting 440 study participants
Last Updated