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Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa
Sponsor: NightstaRx Ltd, a Biogen Company
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
Key Details
Gender
MALE
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
330
Start Date
2018-06-04
Completion Date
2026-06-04
Last Updated
2025-06-12
Healthy Volunteers
No
Interventions
BIIB111
Administered as specified in the treatment arm.
BIIB112
Administered as specified in the treatment arm.
Locations (25)
UCLA - Jules Stein Eye Institute
Los Angeles, California, United States
Vitreo Retinal Associates PA - The Millennium Center
Gainesville, Florida, United States
University of Miami
Miami, Florida, United States
Johns Hopkins Hospital
Baltimore, Maryland, United States
MEEI Massachusets Eye and Ear Infirmary
Boston, Massachusetts, United States
Columbia University Medical Center
New York, New York, United States
Cincinnati Eye Institute - Blue Ash
Cincinnati, Ohio, United States
OHSU - Casey Eye Institute
Portland, Oregon, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
University of Wisconsin School of Medicine
Madison, Wisconsin, United States
Instituto Genetica Ocular
São Paulo, Brazil
The Northern Alberta Clinical Trials and Research Centre
Edmonton, Alberta, Canada
The University of British Columbia - Eye Care Centre
Vancouver, British Columbia, Canada
McGill University Health Centre
Montreal, Quebec, Canada
Rigshospitalet-Glostrup, Oejenafdelingen
Glostrup Municipality, Denmark
Helsinki University Central Hospital (HUCH)
Helsinki, Finland
CHU Montpellier - Saint Eloi
Montpellier, France
Centre Hospitalier National d Ophtalmologie (CHNO) des Quinze-Vingts
Paris, France
Universitäts-Augenklinik Bonn
Bonn, Germany
Universitats Klinikum Tubingen - Institute for Ophthalmic Research
Tübingen, Germany
Radboudumc
Nijmegen, Netherlands
Moorfields Eye Hospital
London, United Kingdom
Manchester Royal Eye Hopsital
Manchester, United Kingdom
John Radcliffe Hospital
Oxford, United Kingdom
Southampton General Hospital
Southampton, United Kingdom