The first-in-human Phase 1/Phase 2a study described herein will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in both healthy participants (HP) and in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Participants who are healthy as determined by medical evaluation
History of recent meningococcal, pneumococcal and Haemophilus influenzae type B vaccinations or willing to be vaccinated
Screening tests negative for illicit drug, nicotine, and alcohol use
History of any significant medical conditions, except for completely excised non-melanoma skin cancer or low grade cervical intraepithelial neoplasia without evidence of recurrence within the prior 3 months
Any viral, bacterial, parasitic, or fungal infection within the prior 30 days
Frequent respiratory, nasopharyngeal or ear infections (more than 5 infections per year)
History of environmental exposure or sick contact that increase the risk of meningococcal, pneumococcal and/or Haemophilus influenza type B infections
ADARx Pharmaceuticals, Inc.
Lead sponsor
ADARx Australia Pty Ltd
Collaborator
Novotech (Australia) Pty Limited
Collaborator