A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111

Overview

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).

Full Title of Study: “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)”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: February 28, 2022

Detailed Description

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

Interventions

  • Drug: BIIB111
    • Administered as specified in the treatment arm.

Arms, Groups and Cohorts

  • Experimental: 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.

Clinical Trial Outcome Measures

Primary Measures

  • Best-Corrected Visual Acuity (BCVA)
    • Time Frame: Up to 2 years
    • BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved.
  • Ophthalmic Examination Assessment: Intraocular Pressure (IOP)
    • Time Frame: Up to 2 years
  • Ophthalmic Examination Assessment: Abnormal Slit Lamp Examination
    • Time Frame: Up to 2 years
  • Ophthalmic Examination Assessment: Dilated Ophthalmoscopy
    • Time Frame: Up to 2 years
  • Ophthalmic Examination Assessment: Lens Opacity Grading
    • Time Frame: Up to 2 years
  • Spectral Domain Optical Coherence Tomography (SD-OCT)
    • Time Frame: Up to 2 years
  • Fundus Autofluorescence (AF)
    • Time Frame: Up to 2 years
  • Fundus Photography
    • Time Frame: Up to 2 years
  • Microperimetry
    • Time Frame: Up to 2 years
  • Percentage of Participants with Adverse Events (AEs)
    • Time Frame: Up to 2 years
  • Vector Shedding Post-treatment
    • Time Frame: Up to 2 years
  • Number of Participants with Cell-Based and Antibody-Based Immune Responses Against BIIB111
    • Time Frame: Up to 2 years
  • Number of Participants with Significant Change in Vital Signs Post-Treatment
    • Time Frame: Up to 2 years

Secondary Measures

  • Change from Baseline in BCVA
    • Time Frame: Up to 2 years
    • BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved.
  • Change from Baseline in AF
    • Time Frame: Up to 2 years
  • Change from Baseline in SD-OCT
    • Time Frame: Up to 2 years
  • Change from Baseline in Microperimetry
    • Time Frame: Up to 2 years

Participating in This Clinical Trial

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

Gender Eligibility: Male

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • NightstaRx Ltd, a Biogen Company
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Medical Director, Study Director, Biogen

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