Phase 1 Study of ADX-626 in Healthy Participants
Research type
Research Study
Full title
A Phase 1, Randomized, Blinded, Placebo-Controlled Study to Assess ADX-626 in Healthy Participants
IRAS ID
1011977
Contact name
Tim Peters-Strickland
Contact email
Sponsor organisation
ADARx Pharmaceuticals, Inc.
Research summary
ADARx Pharmaceuticals is funding this research to evaluate the safety and tolerability of a new drug, ADX-626, for the treatment of conditions with increased risk of thrombosis.
Individuals at high risk of thrombosis include those with a history of blood clots, medical conditions like cancer or atrial fibrillation, and genetic predispositions such as Factor V Leiden mutation. Lifestyle factors like smoking, obesity, prolonged immobility, and hormone therapy also increase this risk.
ADX-626 is a small interfering RNA (siRNA), delivered by subcutaneous injection. It is designed to promote the degradation of FXI in the liver.
FXI is a protein synthesised in the liver which plays a role in enhancing and stabilising the thrombus. Inhibition of FXI prevents thrombosis while minimising bleeding risk. It is well-known that individuals with congenital FXI deficiency experience very low levels of serious bleeding, while known to have a reduced risk of ischemic stroke and venous thromboembolism. Emerging clinical data from several investigational FXI inhibitors show they prevent clots effectively with significantly lower bleeding risk compared to current approved anticoagulants.
This first-in-humans trial in healthy participants aims primarily to look at the safety and tolerability of ADX-626. This clinical trial will also look at how long the drug remains in the body (pharmacokinetics) and how it affects the body (pharmacodynamics).
The study will enrol up to 44 male and female participants of non-childbearing potential in up to 6 sequential dose cohorts. Within each cohort, participants will be randomised and are planned to receive one dose of ADX-626 or placebo. Second dose of ADX-626 or placebo might be added for 1 or two cohorts, preceded by recommendations provided by the Safety Review Committee. The plan duration for participation for each participant is approximately 14-20 months.
REC name
London - London Bridge Research Ethics Committee
REC reference
25/LO/0249
Date of REC Opinion
3 Jun 2025
REC opinion
Further Information Favourable Opinion