This study is accepting new patients by invitation only

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 study on Choroideremia Retinitis Pigmentosa

Summary

Eligibility
for males ages 18 years and up (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
completion around

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 18 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

  • UCLA - Jules Stein Eye Institute
    Los Angeles California 90095-7065 United States
  • OHSU - Casey Eye Institute
    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 330 study participants
Last Updated