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Long-term Safety of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05741346
Recruitment Status : Recruiting
First Posted : February 23, 2023
Last Update Posted : September 25, 2023
Sponsor:
Information provided by (Responsible Party):
BioCryst Pharmaceuticals

Study Description
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Brief Summary:
This study is designed to provide continued access to BCX9930 for subjects currently receiving treatment with BCX9930 in a BioCryst-sponsored clinical study for paroxysmal nocturnal hemoglobinuria (PNH) who, in the opinion of the investigator, would benefit from continued treatment with BCX9930 and who do not have access to other treatment options.

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

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Study to Evaluate the Long-term Safety of BCX9930 Monotherapy in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Previously Received BCX9930 in a BioCryst-sponsored Study
Actual Study Start Date : January 18, 2023
Estimated Primary Completion Date : February 2026
Estimated Study Completion Date : February 2026

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: BCX9930
All subjects receive BCX9930 for 96 weeks
 Drug: BCX9930
Taken orally at 400 mg twice daily (BID)


Outcome Measures
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Primary Outcome Measures :
  1. Proportion of subjects with treatment emergent adverse events (TEAEs) [ Time Frame: Week 96 ]
    Proportion of subjects with TEAEs. Safety assessments include graded TEAEs / serious TEAEs, lab abnormalities, changes to vital signs, and physical examination findings.


Eligibility Criteria
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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
  • Are receiving treatment with BCX9930 in another clinical study of PNH and, in the opinion of the investigator, have benefited from treatment with BCX9930 and would benefit from continued treatment with BCX9930, and who do not have access to other treatment options

Exclusion Criteria:

  • Any clinically significant medical or psychiatric condition including alcohol or drug dependency that, in the opinion of the investigator or sponsor, would interfere with the subject's ability to participate in the study or increase the risk of participation for that subject
  • An ongoing adverse event, including a laboratory abnormality, or other unacceptable toxicity that, in the judgment of the investigator, compromises the ability of the subject to continue study-specific procedures or it is considered not to be in the subject's best interest to continue or benefit-risk assessment is no longer in favor of the subject's continued treatment
Contacts and Locations
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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): NCT05741346


Contacts
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Contact: BioCryst Pharmaceuticals, Inc. +1 (844) 273-2328 BCX9930clinicaltrials@biocryst.com

Locations
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France
Investigative Site Active, not recruiting
Paris, France
Hungary
Investigative Site Active, not recruiting
Budapest, Hungary
Korea, Republic of
Investigative Site Active, not recruiting
Daejeon, Korea, Republic of
Malaysia
Investigative Site Active, not recruiting
Ampang, Malaysia
South Africa
Investigative Site Recruiting
Bloemfontein, South Africa
Investigative Site Active, not recruiting
Cape Town, South Africa
Investigative Site Recruiting
Pretoria, South Africa
Spain
Investigative Site Active, not recruiting
Barcelona, Spain
Investigative Site Active, not recruiting
Valencia, Spain
United Kingdom
Investigative Site Active, not recruiting
Leeds, United Kingdom
Investigative Site Active, not recruiting
London, United Kingdom
Sponsors and Collaborators
BioCryst Pharmaceuticals
Investigators
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Principal Investigator: Phillip Scheinberg, MD, PhD Beneficência Portuguesa de São Paulo
More Information
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Responsible Party: BioCryst Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05741346    
Other Study ID Numbers: BCX9930-205
2021-006776-17 ( EudraCT Number )
First Posted: February 23, 2023    Key Record Dates
Last Update Posted: September 25, 2023
Last Verified: July 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by BioCryst Pharmaceuticals:
BCX9930
Factor D inhibitor
Proximal complement inhibitor
Oral therapy
Paroxysmal nocturnal hemoglobinuria
Additional relevant MeSH terms:
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Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases
 Male Urogenital Diseases
Urological Manifestations
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases