Phase 2 Study of ADX-038 in Participants With Geographic Atrophy
Research type
Research Study
Full title
A Phase 2, Randomized, Masked, Placebo-Controlled Study of Subcutaneously Administered ADX-038 in Participants With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
IRAS ID
1012582
Contact name
Christopher Claeboe
Contact email
Sponsor organisation
ADARx Pharmaceuticals, Inc.
Eudract number
2025-521779-30
ISRCTN Number
Not applicable.
Clinicaltrials.gov Identifier
Research summary
Age-Related Macular Degeneration (AMD) is a common eye condition in older adults that affects the central part of the retina (a light-sensitive tissue at the back of the eye), leading to blurry or reduced central vision. Geographic Atrophy (GA) is an advanced stage of AMD where specific areas of the retina gradually degenerate, resulting in permanent blind spots in central vision. These blind spots enlarge with time and can lead to serious vision loss.
ADX-038 is a new type of treatment known as small interfering RNA (siRNA) therapy. It is designed to use tiny molecules to block the body from making a specific protein called complement factor B (CFB), which is made in the liver. CFB is involved in the body’s immune system and inflammation process. By lowering CFB levels, ADX-038 may help reduce inflammation and prevent further eye damage.
This study will assess the efficacy and safety of ADX-038 for the treatment of GA and also aims to better understand the studied disease and associated health problems. In this study, there will be 2 treatment groups: ADX-038 400mg or placebo. Participants will receive up to 6 doses of ADX-038 or placebo 3 months apart through month 12 and one additional dose at month 18 in the second year, administered by an injection under the skin (subcutaneous). The overall study is expected to take 47 months to complete. Safety eye tests will be performed and blood tests will be performed throughout the study to evaluate whether ADX-038 is working. The test results will be compared to those obtained from participants taking placebo to measure the effectiveness of ADX-038.
This research is being conducted at research sites in the United States, Canada, Australia, United Kingdom and Europe by ADARx Pharmaceuticals Inc. based in the United States.REC name
London - London Bridge Research Ethics Committee
REC reference
25/LO/0845
Date of REC Opinion
9 Dec 2025
REC opinion
Further Information Favourable Opinion