BCX10013

An Open-Label, Multicenter, Intra-Subject Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Therapeutic Potential of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria

Study No: BCX10013

Study Overview: This is a multicenter, open-label, intra-subject, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and therapeutic potential of BCX10013 in participants with paroxysmal nocturnal hemoglobinuria (PNH). Approximately 15 participants will be enrolled in this study. Participants may receive treatment for up to 24 weeks.

Condition/Disease: Paroxysmal Nocturnal Hemoglobinuria (PNH)

Intervention / Treatment: BCX10013 (Drug)

Study Start: 24-10-2023

Enrollment: Ongoing

Study Phase: Phase 1

Investigator site:

Sponsor: BioCryst Pharmaceuticals