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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04702568
Recruitment Status : Enrolling by invitation
First Posted : January 11, 2021
Last Update Posted : January 11, 2021
Sponsor:
Information provided by (Responsible Party):
BioCryst Pharmaceuticals

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria PNH Drug: BCX9930 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 200 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Actual Study Start Date : December 18, 2020
Estimated Primary Completion Date : October 2024
Estimated Study Completion Date : January 2025


Arm Intervention/treatment
Experimental: BCX9930
Intervention: Drug: BCX9930
Drug: BCX9930
BCX9930 for oral administration




Primary Outcome Measures :
  1. Incidence of graded treatment-emergent adverse events [ Time Frame: Week 50 ]
  2. Incidence of graded laboratory chemistry abnormalities [ Time Frame: Week 50 ]
  3. Incidence of graded hematology abnormalities [ Time Frame: Week 50 ]
  4. Incidence of graded coagulation abnormalities [ Time Frame: Week 50 ]
  5. Change in blood pressure [ Time Frame: Week 50 ]
  6. Change in temperature [ Time Frame: Week 50 ]
  7. Change in heart rate [ Time Frame: Week 50 ]
  8. Change in respiratory rate [ Time Frame: Week 50 ]
  9. Change in Electrocardiogram (PR interval) [ Time Frame: Week 50 ]
  10. Change in Electrocardiogram (QT interval) [ Time Frame: Week 50 ]
  11. Change in Electrocardiogram (QRS interval) [ Time Frame: Week 50 ]
  12. Change in Electrocardiogram (RR interval) [ Time Frame: Week 50 ]

Secondary Outcome Measures :
  1. Number of blood transfusions [ Time Frame: Day 1 through Week 50 ]
  2. Lactate dehydrogenase [ Time Frame: absolute and change from Day 1 through Week 50 ]
  3. Hemoglobin [ Time Frame: absolute and change from Day 1 through Week 50 ]
  4. Absolute reticulocyte count [ Time Frame: absolute and change from Day 1 through Week 50 ]
  5. Haptoglobin [ Time Frame: absolute and change from Day 1 through Week 50 ]
  6. Clinical PNH symptom assessments [ Time Frame: absolute and change from Day 1 through Week 50 ]
  7. Serum AP complement activity [ Time Frame: change through Week 50 ]
  8. Plasma Factor Bb [ Time Frame: change through Week 50 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04702568


Locations
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Austria
Study Center
Vienna, Austria
United Kingdom
Study Center
London, United Kingdom
Sponsors and Collaborators
BioCryst Pharmaceuticals
Investigators
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Principal Investigator: Morag Griffin, MBChB Leeds Teaching Hospitals NHS Trust, Leeds, UK
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Responsible Party: BioCryst Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04702568    
Other Study ID Numbers: BCX9930-201
2020-000501-93 ( EudraCT Number )
First Posted: January 11, 2021    Key Record Dates
Last Update Posted: January 11, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by BioCryst Pharmaceuticals:
BCX9930
Factor D inhibitor
PNH
paroxysmal Nocturnal Hemoglobinuria
oral treatment
Additional relevant MeSH terms:
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Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases