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

Study Overview
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Brief Summary
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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.

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

Conditions
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Paroxysmal Nocturnal Hemoglobinuria, PNH

Intervention / Treatment
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* Drug: BCX9930


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BCX9930<br>Intervention: Drug: BCX9930 | Drug: BCX9930<br>* BCX9930 for oral administration<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of graded treatment-emergent adverse events |  | Week 50 | 
| Incidence of graded laboratory chemistry abnormalities |  | Week 50 | 
| Incidence of graded hematology abnormalities |  | Week 50 | 
| Incidence of graded coagulation abnormalities |  | Week 50 | 
| Change in blood pressure |  | Week 50 | 
| Change in temperature |  | Week 50 | 
| Change in heart rate |  | Week 50 | 
| Change in respiratory rate |  | Week 50 | 
| Change in Electrocardiogram (PR interval) |  | Week 50 | 
| Change in Electrocardiogram (QT interval) |  | Week 50 | 
| Change in Electrocardiogram (QRS interval) |  | Week 50 | 
| Change in Electrocardiogram (RR interval) |  | Week 50 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of blood transfusions |  | Day 1 through Week 50 | 
| Lactate dehydrogenase |  | absolute and change from Day 1 through Week 50 | 
| Hemoglobin |  | absolute and change from Day 1 through Week 50 | 
| Absolute reticulocyte count |  | absolute and change from Day 1 through Week 50 | 
| Haptoglobin |  | absolute and change from Day 1 through Week 50 | 
| Clinical PNH symptom assessments |  | absolute and change from Day 1 through Week 50 | 
| Serum AP complement activity |  | change through Week 50 | 
| Plasma Factor Bb |  | change through Week 50 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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oral treatment, BCX9930, PNH, Factor D inhibitor, paroxysmal Nocturnal Hemoglobinuria