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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Paroxysmal Nocturnal Hemoglobinuria; PNH
• Intervention / Treatment: Drug: BCX9930

• Study Type: Interventional
• Enrollment (Anticipated): 200
• Phase: Phase 2

Contacts and Locations

Study Locations

• Austria
  • Vienna, Austria
    • Study Center
• South Africa
  • Bloemfontein, South Africa
    • Study Center
  • Cape Town, South Africa
    • Investigative Site
  • Pretoria, South Africa
    • Study Center
• United Kingdom
  • London, United Kingdom
    • Study Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BCX9930; Intervention: Drug: BCX9930	Drug: BCX9930; BCX9930 for oral administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: BioCryst Pharmaceuticals
• Investigators: Principal Investigator: Morag Griffin, MBChB, Leeds Teaching Hospitals NHS Trust, Leeds, UK

Study record dates

Study Major Dates

• Study Start (Actual): December 18, 2020
• Primary Completion (Anticipated): October 1, 2024
• Study Completion (Anticipated): January 1, 2025

Study Registration Dates

• First Submitted: January 7, 2021
• First Submitted That Met QC Criteria: January 8, 2021
• First Posted (Actual): January 11, 2021

Study Record Updates

• Last Update Posted (Actual): April 25, 2023
• Last Update Submitted That Met QC Criteria: April 24, 2023
• Last Verified: April 1, 2023

More Information

Terms related to this study

• Keywords: PNH; Factor D inhibitor; BCX9930; paroxysmal Nocturnal Hemoglobinuria; oral treatment
• Additional Relevant MeSH Terms: Urologic Diseases; Urological Manifestations; Bone Marrow Diseases; Hematologic Diseases; Urination Disorders; Anemia; Proteinuria; Anemia, Hemolytic; Myelodysplastic Syndromes; Hemoglobinuria; Hemoglobinuria, Paroxysmal
• Other Study ID Numbers: BCX9930-201; 2020-000501-93 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No