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
Parallel
ADX-038
DrugsiRNA duplex oligonucleotide
Placebo
DrugSaline
Safety in Healthy Volunteers
To evaluate the safety and tolerability of ADX-038 in HVs by incidence, relationship, and severity of adverse events and serious adverse events
Safety in Paroxysmal Nocturnal Hemoglobinuria Participants
Evaluate the safety and tolerability of ADX-038 by incidence, relationship, and treatment-emergent adverse events and serious adverse events.
Pharmacokinetics in Healthy Participants
To characterize the Pharmacokinetics of ADX-038 in HPs by measuring the Maximum observed plasma concentration (Cmax)
Pharmacodynamics in Healthy Participants
Change from base in plasma concentrations over time in Complement factor B (CFB) protein via assay measurement
Pharmacodynamics in Paraxysmal Nocturnal Hemoglobinuria
Evaluate the changes in hemoglobin concentrations
Pharmacodynamics in Paraxysmal Nocturnal Hemoglobinuria
Characterize the changes in serum concentration of complement factor B (CFB) protein and complement alternative pathway activity level.
Immune Response in Healthy Participants
Anti-polyethylene glycol (PEG) antibody titers and anti-drug antibody titers
Pharmacodynamics in Healthy Participants
Change in complement classical pathway activity via assay measurement
Safety in Paroxysmal Nocturnal Hemoglobinuria Participants
Evaluate the change from baseline in follow up visits for safety assesments
Clinical Effect in Paroxysmal Nocturnal Hemoglobinuria Participants
Look at time (number of days) from dosing (Day 1) to diminished therapeutics effect (DTE)
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ADARx Pharmaceuticals, Inc.
Lead sponsor
ADARx Australia Pty Ltd
Collaborator
Novotech (Australia) Pty Limited
Collaborator
