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)
Verified date | May 2024 |
Source | BioCryst Pharmaceuticals |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
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 |
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 |
Lead Sponsor | Collaborator |
---|---|
BioCryst Pharmaceuticals |
Austria, South Africa, United Kingdom,
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 |
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