A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111
NCT ID: NCT03507686
Last Updated: 2024-02-23
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
66 participants
INTERVENTIONAL
2017-11-29
2022-06-29
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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BIIB111
Participants will receive a single dose of sub-retinal injection of BIIB111 in each eye at Day 0 separated by an interval of \<6 months, 6-12 months, or \>12 months.
BIIB111
Administered as specified in the treatment arm.
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
2. Have documentation of a genetically-confirmed diagnosis of CHM.
3. Have active disease clinically visible within the macular region of both eyes.
4. Have a BCVA of ≥34 ETDRS letters (20/200 or better Snellen acuity) in both eyes, or in the untreated eye, if the other eye was previously treated with BIIB111\*
\*If previously treated with BIIB111 in an antecedent study, participants may be eligible for participation following Sponsor approval.
5. For participants who received treatment with BIIB111 in an antecedent study, have biological samples available to complete an adequate immunology profile.
Exclusion Criteria
2. Are unwilling to use barrier contraception methods or abstain from sexual intercourse for a period of 3 months following treatment with BIIB111 in either eye.
3. Have had previous intraocular surgery performed within 3 months of the Screening Visit in either eye.
4. 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. This includes but is not limited to a potential participants:
* with a contraindication to oral corticosteroid (e.g., prednisolone/prednisone)
* with clinically significant cataract in either eye
* who, in the clinical opinion of the Investigator, is not an appropriate candidate for sub-retinal surgery.
5. 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, except if treated within an antecedent study with BIIB111.
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
Miami, Florida, United States
Research Site
Boston, Massachusetts, United States
Research Site
Cincinnati, Ohio, United States
Research Site
Portland, Oregon, United States
Research Site
Paris, , France
Research Site
Tübingen, , Germany
Countries
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References
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MacLaren RE, Audo I, Fischer MD, Huckfeldt RM, Lam BL, Pennesi ME, Sisk R, Gow JA, Li J, Zhu K, Tsang SF. An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study. Hum Gene Ther. 2024 Aug;35(15-16):564-575. doi: 10.1089/hum.2024.017. Epub 2024 Jul 27.
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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2017-002395-75
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
273CH203
Identifier Type: -
Identifier Source: org_study_id
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