Clinical trial NCT04702568: A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Official title: A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Paroxysmal Nocturnal Hemoglobinuria
PNH
Keywords: BCX9930
Factor D inhibitor
PNH
paroxysmal Nocturnal Hemoglobinuria
oral treatment
Lead sponsor: BioCryst Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2021-01-11
Enrollment: 19
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

* Male or non-pregnant, non-lactating female participants
* 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 participant's ability to participate in the study or participation would increase the risk for that participant
* Pregnant, planning to become pregnant, or having been pregnant within 90 days of Day 1, or lactating

Note: Other protocol-defined Inclusion/Exclusion criteria may apply

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
timeFrame: From first dose of study drug up to 3 weeks after last dose (Week 147)
Secondary outcomes:
measure: Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue
timeFrame: Baseline, Weeks 24, 48, 72, 96, and 120
measure: Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea
timeFrame: Baseline, Weeks 24, 48, 72, 96, and 120
measure: Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort
timeFrame: Baseline, Weeks 24, 48, 72, 96, and 120
measure: Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing
timeFrame: Baseline, Weeks 24, 48, 72, 96, and 120
measure: Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain
timeFrame: Baseline, Weeks 24, 48, 72, 96, and 120
measure: Number of Participants With Clinical PNH Symptom Based on Severity: Headache
timeFrame: Baseline, Weeks 24, 48, 72, 96, and 120
measure: Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction
timeFrame: Baseline, Weeks 24, 48, 72, 96, and 120
measure: Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria
timeFrame: Baseline, Weeks 24, 48, 72, 96, and 120
measure: Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice
timeFrame: Baseline, Weeks 24, 48, 72, 96, and 120
measure: Change From Baseline in Lactate Dehydrogenase (LDH)
timeFrame: Baseline, Weeks, 24, 48, 72, 96, 120, and 144
measure: Change From Baseline in Hemoglobin
timeFrame: Baseline, Weeks 24, 48, 72, 96, 120, and 144
measure: Change From Baseline in Haptoglobin
timeFrame: Baseline, Weeks 24, 48, 72, 96, and 120
measure: Change From Baseline in Reticulocytes
timeFrame: Baseline, Weeks 24, 48, 72, 96, and 120
measure: Number of Participants With Blood Transfusions or Thromboses
timeFrame: From first dose of study drug up to 3 weeks after last dose (Week 147)
measure: Number of Blood Transfusions
timeFrame: From first dose of study drug up to 3 weeks after last dose (Week 147)
measure: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score
timeFrame: Baseline, Weeks 24, 48, 72, and 96
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: Study Center
city: Bloemfontein
country: South Africa
facility: Study Center
city: Cape Town
country: South Africa
facility: Study Center
city: Pretoria
country: South Africa
facility: Study Center
city: London
country: United Kingdom
facility: Study Center
Study officials:
affiliation: Leeds Teaching Hospitals NHS Trust, Leeds, UK
name: Morag Griffin, MBChB
role: PRINCIPAL_INVESTIGATOR
References: