Clinical trial
An Open-Label Safety Study of Retinal Gene Therapy for Choroideremia With Bilateral, Sequential Administration of Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)
Name
273CH203
Description
The objective of the study is to evaluate the safety of bilateral, sequential sub-retinal administration of a single dose of BIIB111 in adult male participants with Choroideremia (CHM).
Trial arms
Trial start
2017-11-29
Estimated PCD
2022-06-29
Trial end
2022-06-29
Status
Completed
Phase
Early phase I
Treatment
BIIB111
Administered as specified in the treatment arm.
Arms:
BIIB111
Other names:
Gene Therapy, AAV2-REP1
Size
66
Primary endpoint
Mean Best-Corrected Visual Acuity (BCVA) as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart in Letters at Month 12
Month 12
Ophthalmic Examination Assessment: Mean Intraocular Pressure (IOP) at Month 12
Month 12
Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Slit Lamp Examination
Baseline, Month 12
Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Dilated Ophthalmoscopy
Baseline, Month 12
Ophthalmic Examination Assessment: Number of Participants With Lens Opacity Grading
Month 12
Spectral Domain Optical Coherence Tomography (SD-OCT): Foveal Subfield Thickness at Month 12
Month 12
SD-OCT: Total Macular Volume at Month 12
Month 12
SD-OCT: Central Horizontal Ellipsoid Width at Month 12
Month 12
SD-OCT: Central Ellipsoid Area at Month 12
Month 12
SD-OCT: Square Root of Central Ellipsoid Area at Month 12
Month 12
SD-OCT: Choroidal Thickness at Foveal Center at Month 12
Month 12
Fundus Autofluorescence (AF): Mean Total Area of Preserved Autofluorescence at Month 12
Month 12
AF: Mean Square Root of Total Area of Preserved AF at Month 12
Month 12
AF: Mean Distance From Foveal Center to Nearest Border of Preserved AF at Month 12
Month 12
Fundus Photography: Number of Participants With Retinal Pigment Epithelium (RPE) Hyperplasia as Per Severity
Month 12
Fundus Photography: Number of Participants With Retinal Arteriolar Narrowing as Per Severity
Month 12
Fundus Photography: Number of Participants With Retinal Vessel Sheathing as Per Severity
Month 12
Fundus Photography: Number of Participants With Optic Atrophy/Pallor as Per Severity
Month 12
Fundus Photography: Number of Participants With Optic Disc Swelling as Per Severity
Month 12
Microperimetry: Retinal Mean Sensitivity at Month 12
Month 12
Microperimetry: Bivariate Contour Ellipse Area 63% at Month 12
Month 12
Microperimetry: Bivariate Contour Ellipse Area 95% at Month 12
Month 12
Microperimetry: Fixation Losses (in Percentage) at Month 12
Month 12
Percentage of Participants With at Least One Treatment-Emergent Adverse Event (TEAE)
Day 0 (surgery) in period 1 up to last follow up visit in period 2 (approximately 2 years)
Number of Participants With Vector Shedding Post-treatment at Month 3
Baseline, at Month 3
Number of Participants With Anti-drug Antibodies Post-treatment at Month 12
Month 12
Vital Signs: Change From Baseline in Blood Pressure at Month 12
Baseline, Month 12
Vital Signs: Change From Baseline in Pulse Rate at Month 12
Baseline, Month 12
Eligibility criteria
Key Inclusion Criteria:
1. Are willing and able to give informed consent for participation in the study to have both eyes treated.
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.
Key Exclusion Criteria:
1. Have a history of amblyopia or inflammatory disorder in either eye.
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.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 66, 'type': 'ACTUAL'}}
Updated at
2024-02-23
1 organization
1 product
1 indication
Organization
BiogenProduct
BIIB111Indication
Choroideremia