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 PNH. Approximately 8 participants will be enrolled in this study. Participants may receive treatment for up to 52 weeks.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
Multiple dose levels may be tested in this study.
BioCryst Investigative Site
Ampang, Malaysia
BioCryst Investigative Site
Bloemfontein, South Africa
BioCryst Investigative Site
Cape Town, South Africa
BioCryst Investigative Site
Pretoria, South Africa
Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Graded Laboratory Abnormalities, and Changes From Baseline (CFB) in Laboratory Analytes, Vital signs, Electrocardiograms (ECGs), and Physical Examination Findings.
Time frame: up to 52 weeks
CFB in Lactate Dehydrogenase
Time frame: Baseline, Week 52
CFB in the Ratio of Total PNH Red Blood Cell Clone Size to PNH White Blood Cell Clone Size
Time frame: Baseline, Week 52
CFB in Hemoglobin
Time frame: Baseline, Week 52
Percentage of Participants who are Transfusion-free
Time frame: 52 weeks
Percentage of Participants Achieving a Within-subject Clinically Meaningful CFB in the FACIT-Fatigue scale
Time frame: 52 weeks
CFB in Other Clinical Biomarkers of PNH Disease Activity including absolute reticulocyte count, total PNH red blood cell clone size, haptoglobin levels, total bilirubin, and aspartate transaminase
Time frame: Baseline, Week 52
Number of Participants with Clinical PNH Symptoms
Time frame: up to 52 weeks
Concentration of BCX10013 and its Metabolite(s) in Plasma
Time frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post dose on Day 1, Week 2, and Week 4
Concentration of BCX10013 and its Metabolite(s) in Urine (if applicable)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time frame: Pre-dose and all urine from 0 to 6 hours post dose on Day 1, Week 2, and Week 4