Oral Berotralstat Expanded Access Program

NCT04428632 | Unknown

• 1 other identifier: BCX7353-308
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This expanded access program will provide access to berotralstat for eligible participants with hereditary angioedema in the U.S.

Conditions

Hereditary Angioedema; HAE; Prophylaxis

Keywords

Berotralstat; BCX7353; Hereditary Angioedema; HAE

Interventions

Berotralstat DRUG

One 150mg capsule administered orally once daily

Also known as: BCX7353

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• BioCryst Pharmaceuticals: lead

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

berotralstat

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 10, 2020
• First Posted: June 11, 2020
• Last Updated: December 11, 2020

Record last verified: 2020-12