A Phase 1/2 Study of the Safety and Efficacy of BRX011 Oral Administration Once Daily in Subjects With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Overview

This study aims to evaluate the safety and efficacy of BRX011, an oral medication, taken once daily by participants with geographic atrophy secondary to age-related macular degeneration. The study is conducted in phases 1 and 2, focusing on assessing both safety (tolerability) and effectiveness (efficacy) of the treatment. Participants: Adults with geographic atrophy due to age-related macular degeneration. Treatment: BRX011 or Placebo is taken once daily as per the protocol. Duration: The study involves multiple visits over 96 weeks to monitor participants' health and response to treatment. Safety Monitoring: Regular checks for adverse events and health status to ensure participant well-being. Checks will include examination of vital signs, clinical labs, ocular exams, and ocular imaging. Primary Outcome Measure: Efficacy of BRX011 in the annual rate of change in the square root of GA area, as specified in the protocol.

Interventions

• Drug BRX011
  Subjects will ingest BRX011 orally, as capsules.
• Drug Placebo
  Subjects will ingest Placebo orally, as capsules.

Primary outcome measures

• Annual rate of change in the square root of GA area [Time frame: Baseline (Day 0) to Week 96]

Secondary outcome measures (2)

• Change in low luminance deficit [Time frame: Baseline (Day 0) to Week 96]
• Change in ETDRS BCVA [Time frame: Baseline (Day 0) to Week 96]

Eligibility criteria

Inclusion criteria

• Age ≥55 years
• Visual acuity in the study eye at Screening/Baseline

• BCVA ≥35 letters using ETDRS charts (≥20/200 Snellen equivalent)
• LLD >5 letters
• Clinical diagnosis of GA secondary to AMD. CNV in the fellow eye is permitted.

(5) Clarity of ocular media, adequate pupillary dilation, and fixation to permit the evaluation of the eye, as determined by the investigator

(6) Female subjects must be of non-child-bearing potential (WONCBP), defined as:

(a) A woman who had surgical sterilization (hysterectomy, bilateral oophorectomy, or bilateral salpingectomy) (b) A woman ≥ 60 years of age (c) A woman ≥ 55 and < 60 years of age who fulfills at least one of the following: (i) A cessation of menses for at least 12 months and a folliclestimulating hormone (FSH) test confirming non-childbearing potential (refer to laboratory reference ranges for confirmatory levels) (ii) A cessation of menses for at least 24 months without FSH levels confirmed.

(7) Willing and able to give informed consent and to comply with the study procedures and assessments

Exclusion criteria

• GA secondary to a condition other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like plaquenil maculopathy in either eye
• Any history documented or active CNV
• Any ocular condition other than GA secondary to AMD that may require surgery or medical intervention during the study period

(5) History of vitrectomy surgery, submacular surgery, other surgical intervention for AMD, corneal transplant, glaucoma filtration surgery, or cataract surgery within 3 months prior to Screening/Baseline

(6) History of intravitreal injection of anti-vascular endothelial growth factor (VEGF) therapies in the study eye at any time, history of intravitreal injection of any agent (e.g., triamcinolone) in the study eye within the last 3 months prior to study enrollment. A single intraoperative administration of a corticosteroid during cataract surgery at least 3 months prior to Screening is permitted

(7) History of laser therapy in the macular region in the study eye, prior treatment with photobiomodulation, external-beam radiation therapy or transpupillary thermotherapy in study eye

(8) Previous cell-based intraocular treatment in the study eye or previous expression vector-mediated intraocular treatments in either eye (i.e., gene therapy), any previous treatment with any deuterated molecules for eye diseases (e.g., deuterated vitamin A)

(9) History of idiopathic or autoimmune-associated uveitis, ocular or intraocular conditions, and infectious or inflammatory ocular disease. Active uveitis and infectious conjunctivitis, keratitis, scleritis or endophthalmitis

(11) Active malignancy within the previous 12 months except for appropriately treated carcinoma in situ of cervix, resolved non-melanoma skin carcinoma, and prostate cancer with a Gleason score of less than or equal to (≤) 6, and a stable prostate-specific antigen for greater than or equal to (≥) 12 months

(13) Intake of omega-3 supplements (e.g., fish oil, cod liver oil, krill oil, edible algae oil, flax oil) or prescription omega-3 drugs (e.g., Lovaza® , Vascepa® , Epanova® ) in the past 4 weeks prior to Screening and throughout the duration of the study; intake of supplements containing Lutein, zeaxanthin and or mesozeaxanthin (e.g., Ocuvite® , PreserVision® , SYSTANE ICAPS ® or other AREDS supplements) in the past 4 weeks prior to Screening and throughout the duration of the study

(14) Participation in an interventional clinical study within the past 30 days of Screening, or interventional GA studies within the past 5 months prior to Screening

(15) Treatment with SYFOVRE ®or IZERVAY ®within 3 months prior to Screening

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Triple blind

Primary purpose

Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07029945 · BRX011-001

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗