Efficacy and Safety of BIIB111 for the Treatment of Choroideremia
NCT ID: NCT03496012
Last Updated: 2023-12-07
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
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COMPLETED
PHASE3
169 participants
INTERVENTIONAL
2017-12-11
2020-12-01
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
SINGLE
Study Groups
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BIIB111 High Dose
Participants will receive a single administration of high dose BIIB111 in one eye through sub-retinal injection after vitrectomy.
BIIB111
Administered as specified in the treatment arm.
BIIB111 Low Dose
Participants will receive a single administration of low dose BIIB111 in one eye through sub-retinal injection after vitrectomy.
BIIB111
Administered as specified in the treatment arm.
Untreated Control Group
Participants will receive no sham surgery or study medication.
No interventions assigned to this group
Interventions
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BIIB111
Administered as specified in the treatment arm.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Have a documented genetically-confirmed diagnosis of CHM.
* Have active disease clinically visible within the macular region in the study eye.
* Have a BCVA of 34-73 ETDRS letters (equivalent to worse than or equal to 6/12 or 20/40 Snellen acuity, but better than or equal to 6/60 or 20/200 Snellen acuity) in the study eye.
Exclusion Criteria
* Have had previous intraocular surgery performed in the study eye within 3 months of Visit 1.
* Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may either put the participants at risk because of participation in the study, or may influence the results of the study, or the participant's ability to participate in the study.
* Have participated in another research study involving an investigational product in the past 12 weeks or received a gene/cell-based therapy at any time previously.
* Are unwilling to use barrier contraception methods, or abstain from sexual intercourse, for a period of 3 months, if treated with AAV2-REP1.
18 Years
MALE
No
Sponsors
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Biogen
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Biogen
Locations
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Research Site
Los Angeles, California, United States
Research Site
Miami, Florida, United States
Research Site
Baltimore, Maryland, United States
Research Site
New York, New York, United States
Research Site
Cincinnati, Ohio, United States
Research Site
Portland, Oregon, United States
Research Site
Dallas, Texas, United States
Research Site
Madison, Wisconsin, United States
Research Site
Montreal, , Canada
Research Site
Vancouver, , Canada
Research Site
Glostrup Municipality, , Denmark
Research Site
Helsinki, , Finland
Research Site
Montpellier, , France
Research Site
Bonn, , Germany
Research Site
Tübingen, , Germany
Research Site
Nijmegen, , Netherlands
Research Site
Manchester, , United Kingdom
Research Site
Oxford, , United Kingdom
Countries
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References
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Davis JL. The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases. Am J Ophthalmol. 2018 Dec;196:xxv-xxix. doi: 10.1016/j.ajo.2018.08.038. Epub 2018 Sep 5.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2015-003958-41
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
273CH301
Identifier Type: -
Identifier Source: org_study_id