This study was designed to evaluate the long-term safety of daily oral treatment with BCX9930 in participants who had participated in a previous BCX9930 trial for PNH and showed a benefit of treatment as determined by the Investigator. The study allowed continued access to BCX9930 for enrolled participants. The study also evaluated the long-term effectiveness and impact on quality of life and general well-being of BCX9930 treatment, and the participant's satisfaction with the medication.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
19
BCX9930 for oral administration
Administered at stable dose at the time of study entry
Administered at stable dose at the time of study entry
Study Center
Vienna, Austria
Study Center
Bloemfontein, South Africa
Study Center
Cape Town, South Africa
Study Center
Pretoria, South Africa
Study Center
London, United Kingdom
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An AE was defined as any untoward medical occurrence associated with the use of an intervention in humans, whether or not considered intervention-related in a participant or clinical investigation participant who administered a pharmaceutical product regardless of its causal relationship to the study treatment. An AE was considered treatment-emergent if its start date and time was on or after the date and time of first on-study dose of study drug.
Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue
Data was reported for number of participants with clinical PNH symptom of fatigue. The severity of clinical PNH symptom of fatigue was graded as none, mild, moderate and severe based solely on investigator's discretion.
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea
Data was reported for number of participants with clinical PNH symptom of Dyspnea. The severity of clinical PNH symptom of Dyspnea was graded as none, mild, moderate and severe based solely on investigator's discretion.
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort
Data was reported for number of participants with clinical PNH symptom of Chest pain/discomfort. The severity of clinical PNH symptom of chest pain/discomfort was graded as none, mild, moderate and severe based solely on investigator's discretion.
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing
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Data was reported for number of participants with clinical PNH symptom of difficulty swallowing. The severity of clinical PNH symptom of difficulty swallowing was graded as none, mild, moderate and severe based solely on investigator's discretion.
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain
Data was reported for number of participants with clinical PNH symptom of abdominal pain. The severity of clinical PNH symptom of abdominal pain was graded as none, mild, moderate and severe based solely on investigator's discretion.
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Headache
Data was reported for number of participants with clinical PNH symptom of headache. The severity of clinical PNH symptom of headache was graded as none, mild, moderate and severe based solely on investigator's discretion.
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction
Data was reported for number of participants with clinical PNH symptom of erectile dysfunction. The severity of clinical PNH symptom of erectile dysfunction was graded as none, mild, moderate and severe based solely on investigator's discretion.
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria
Data was reported for number of participants with clinical PNH symptom of Hemoglobinuria.The severity of clinical PNH symptom of hemoglobinuria was graded as none, mild, moderate and severe based solely on investigator's discretion.
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice
Data was reported for number of participants with clinical PNH symptom of jaundice. The severity of clinical PNH symptom of jaundice was graded as none, mild, moderate and severe based solely on investigator's discretion.
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Change From Baseline in Lactate Dehydrogenase (LDH)
Time frame: Baseline, Weeks, 24, 48, 72, 96, 120, and 144
Change From Baseline in Hemoglobin
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, and 144
Change From Baseline in Haptoglobin
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Change From Baseline in Reticulocytes
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Blood Transfusions or Thromboses
Data was reported for number of participants for whom blood transfusion was required or who experienced the thrombosis events.
Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)
Number of Blood Transfusions
Number of blood transfusions were reported.
Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score
The FACIT-Fatigue scale questionnaire was used to determine the level of fatigue experienced by participants. This questionnaire was a 13-item measure that assessed self-reported fatigue and its impact upon daily activities and function. Item scores ranged from 0 ("not at all") to 4 ("very much"), and the total score ranged from 0 to 52, with higher scores indicating greater quality of life.
Time frame: Baseline, Weeks 24, 48, 72, and 96