Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa
NCT ID: NCT03584165
Last Updated: 2025-06-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ENROLLING_BY_INVITATION
PHASE3
330 participants
INTERVENTIONAL
2018-06-04
2026-06-04
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
OTHER
SINGLE
Study Groups
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BIIB111
Participants previously treated with sub-retinal injection of BIIB111 in antecedent studies 273CH301 (NCT03496012) and 273CH203 (NCT03507686) will be enrolled. Participants previously treated with this same sub-retinal injection (rAAV2-REP1) in antecedent studies 20150371 (NCT02553135), Pro00028599 (NCT02077361), THOR-TUE-01 (NCT02671539), CHM09/01 (NCT01461213) and REGEN2015 (NCT02407678) will also be invited for enrollment. This is a follow-up study, investigational product was administered in the previous study.
BIIB111
Administered as specified in the treatment arm.
BIIB112
Participants previously treated with sub-retinal injection of BIIB112 in the antecedent study 274RP101 (NCT03116113) will be enrolled. This is a follow-up study, investigational product was administered in the previous study.
BIIB112
Administered as specified in the treatment arm.
Untreated
Untreated participants who served as controls in the antecedent study 273CH301 (NCT03496012), investigating treatment with BIIB111, will be enrolled. This is a follow-up study, participants will not be administered study medication nor receive a sham surgery.
No interventions assigned to this group
Interventions
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BIIB111
Administered as specified in the treatment arm.
BIIB112
Administered as specified in the treatment arm.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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.
Exclusion Criteria
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.
18 Years
MALE
No
Sponsors
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NightstaRx Ltd, a Biogen Company
OTHER
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Biogen
Locations
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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
Countries
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Other Identifiers
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2017-003104-42
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
273CH201
Identifier Type: -
Identifier Source: org_study_id
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