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Oral Berotralstat Expanded Access Program

Primary Purpose

Hereditary Angioedema, HAE, Prophylaxis

Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
Berotralstat
Sponsored by
BioCryst Pharmaceuticals
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Hereditary Angioedema focused on measuring Berotralstat, BCX7353, Hereditary Angioedema, HAE

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All Sexes

Inclusion Criteria

Patients must meet all of the following inclusion criteria to be eligible for participation in this program:

  1. At least 12 years of age
  2. Able to provide written, informed consent or assent
  3. Patients with a clinical diagnosis of HAE Type I or II who, in the opinion of their treating physician, are expected to benefit from an oral treatment for the prevention of angioedema attacks, and who are not eligible or able to access a berotralstat clinical trial
  4. Females must use acceptable effective contraception

Exclusion Criteria

Patients must meet none of the below exclusion criteria to be eligible for participation in this program:

  1. Pregnancy or breast-feeding
  2. Any clinically significant medical condition or medical history that, in the opinion of the treating physician, would interfere with the patient's safety
  3. Current infection with hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    June 10, 2020
    Last Updated
    December 9, 2020
    Sponsor
    BioCryst Pharmaceuticals
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04428632
    Brief Title
    Oral Berotralstat Expanded Access Program
    Official Title
    Expanded Access Program With Oral Berotralstat for the Prevention of Attacks in Patients With Hereditary Angioedema
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    December 2020
    Overall Recruitment Status
    Approved for marketing
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    BioCryst Pharmaceuticals

    4. Oversight

    5. Study Description

    Brief Summary
    This expanded access program will provide access to berotralstat for eligible participants with hereditary angioedema in the U.S.
    Detailed Description
    Physicians may request access to berotralstat for eligible participants with unmet medical need Physicians can send email inquiries to access.us@inceptua.com or call 1-888-225-8677 Patients who may be interested should contact their physician about participation Berotralstat (BCX7353) will be available through this expanded access program until it is commercially available

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Hereditary Angioedema, HAE, Prophylaxis
    Keywords
    Berotralstat, BCX7353, Hereditary Angioedema, HAE

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    Berotralstat
    Other Intervention Name(s)
    BCX7353
    Intervention Description
    One 150mg capsule administered orally once daily

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    12 Years
    Eligibility Criteria
    Inclusion Criteria Patients must meet all of the following inclusion criteria to be eligible for participation in this program: At least 12 years of age Able to provide written, informed consent or assent Patients with a clinical diagnosis of HAE Type I or II who, in the opinion of their treating physician, are expected to benefit from an oral treatment for the prevention of angioedema attacks, and who are not eligible or able to access a berotralstat clinical trial Females must use acceptable effective contraception Exclusion Criteria Patients must meet none of the below exclusion criteria to be eligible for participation in this program: Pregnancy or breast-feeding Any clinically significant medical condition or medical history that, in the opinion of the treating physician, would interfere with the patient's safety Current infection with hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)

    12. IPD Sharing Statement

    Learn more about this trial

    Oral Berotralstat Expanded Access Program

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