Principal Investigator
Enrollment Status
Clinical Trial ID
Clinical Trial Summary
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).
Phase
Phase 2
Funding Agency/Sponsor
Industry
Disease
Neuro-Ophthalmology
Enrollment Eligibility
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