Efficacy and Safety of BIIB111 for the Treatment of Choroideremia (STAR)

Biogen

Status and phase: Completed; Phase 3

Conditions: Choroideremia

Treatments: Genetic: BIIB111

Study type: Interventional

Funder types: Industry

Identifiers

NCT03496012

273CH301

2015-003958-41 (EudraCT Number)

Details and patient eligibility

About

The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of BIIB111 in participants with choroideremia (CHM).

Full description

This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.

Enrollment: 169 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Are willing and able to give informed consent for participation in the study.
• 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.

Key Exclusion Criteria:

• Have a history of amblyopia in the eligible eye.
• 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.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

---

169 participants in 3 patient groups

BIIB111 High Dose

Experimental group

Description:

Participants will receive a single administration of high dose BIIB111 in one eye through sub-retinal injection after vitrectomy.

Treatment:

Genetic: BIIB111

BIIB111 Low Dose

Experimental group

Description:

Participants will receive a single administration of low dose BIIB111 in one eye through sub-retinal injection after vitrectomy.

Treatment:

Genetic: BIIB111

Untreated Control Group

No Intervention group

Description:

Participants will receive no sham surgery or study medication.