A Long Term Safety Study of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

Official title:

A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04702568
• Other study ID #: BCX9930-201
• Secondary ID: 2020-000501-93
• Status: Terminated
• Phase: Phase 2
• Start date: December 18, 2020
• Est. completion date: October 4, 2023

Study information

• Verified date: May 2024
• Source: BioCryst Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study is designed to evaluate the long-term safety of daily oral treatment with BCX9930 in subjects who have participated in a previous BCX9930 trial for PNH and showed a benefit of treatment as determined by the Investigator. The study allows continued access to BCX9930 for enrolled subjects. The study will also evaluate the long-term effectiveness and impact on quality of life and general well-being of BCX9930 treatment, and the subject's satisfaction with the medication.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 19
• Est. completion date: October 4, 2023
• Est. primary completion date: October 4, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Male or non-pregnant, non-lactating female subjects

• Successfully participated in a previous BCX9930 study of PNH and experienced improvement in their PNH

Exclusion Criteria:

• Apart from a diagnosis of PNH, any clinically significant medical or psychiatric condition or medical history, other than those associated with PNH disease, that, in the opinion of the Investigator or Sponsor, would interfere with the subject's ability to participate in the study or participation would increase the risk for that subject

• Pregnant, planning to become pregnant, or having been pregnant within 90 days of Day 1, or lactating

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria
• PNH

Intervention

Drug:
• BCX9930
BCX9930 for oral administration

Locations

Country	Name	City	State
Austria	Study Center	Vienna
South Africa	Study Center	Bloemfontein
South Africa	Investigative Site	Cape Town
South Africa	Study Center	Pretoria
United Kingdom	Study Center	London

Sponsors (1)

Lead Sponsor	Collaborator
BioCryst Pharmaceuticals

Countries where clinical trial is conducted

Austria,  South Africa,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of graded treatment-emergent adverse events		Week 50
Primary	Incidence of graded laboratory chemistry abnormalities		Week 50
Primary	Incidence of graded hematology abnormalities		Week 50
Primary	Incidence of graded coagulation abnormalities		Week 50
Primary	Change in blood pressure		Week 50
Primary	Change in temperature		Week 50
Primary	Change in heart rate		Week 50
Primary	Change in respiratory rate		Week 50
Primary	Change in Electrocardiogram (PR interval)		Week 50
Primary	Change in Electrocardiogram (QT interval)		Week 50
Primary	Change in Electrocardiogram (QRS interval)		Week 50
Primary	Change in Electrocardiogram (RR interval)		Week 50
Secondary	Number of blood transfusions		Day 1 through Week 50
Secondary	Lactate dehydrogenase		absolute and change from Day 1 through Week 50
Secondary	Hemoglobin		absolute and change from Day 1 through Week 50
Secondary	Absolute reticulocyte count		absolute and change from Day 1 through Week 50
Secondary	Haptoglobin		absolute and change from Day 1 through Week 50
Secondary	Clinical PNH symptom assessments		absolute and change from Day 1 through Week 50
Secondary	Serum AP complement activity		change through Week 50
Secondary	Plasma Factor Bb		change through Week 50