A Phase 1, Randomized, Double-Blind, Placebo-Controlled Study in Healthy Participants Followed by a Phase 2a Open-Label Study in PNH Patients to Evaluate Safety, Tolerability, PK, and PD of ADX-038

What this Trial Is About

Researchers are studying ADX-038, an siRNA duplex oligonucleotide, in a Phase 1/Phase 2a clinical trial to evaluate its safety, tolerability, pharmacokinetics (how the drug moves through the body), and pharmacodynamics (the drug's effects) in both healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The trial is sponsored by ADARx Pharmaceuticals, Inc. and involves both healthy participants and PNH patients who have residual anemia despite standard anti-C5 treatment. The study has two parts: Phase 1 involves a randomized, double-blind, placebo-controlled, single ascending dose (SAD) design in healthy participants across up to 5 dose levels. Each cohort includes 8 participants, with 6 receiving ADX-038 and 2 given placebo. Sentinel participants are initially dosed to monitor safety before the rest are treated. Phase 2a is an open-label, single-arm study where participants with PNH receive ADX-038 at doses determined from Phase 1 results while continuing their standard anti-C5 regimen. Participants will undergo medical evaluations to assess safety over 365 days, including monitoring for side effects and laboratory tests. Pharmacokinetic measurements in healthy participants will be collected over 8 days, while pharmacodynamic effects will be observed for up to one year in both healthy volunteers and PNH patients. The study includes vaccinations against certain infections, screening for infections and medical conditions, and adherence to prophylactic antibiotic use. Total participation time varies by study phase and individual progress.

Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients