Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

This 3-part study will evaluate the efficacy and safety of an oral kallikrein inhibitor, BCX7353, in the treatment angioedema attacks in subjects with Type I or II hereditary angioedema (HAE). In each study part, subjects will treat 3 attacks with BCX7353 (2 attacks) or placebo (1 attack), in a randomly allocated order. In Part 1, the dose of 750mg will be assessed relative to placebo in up to 36 patients. If this is shown to be effective, then a further 12 patients will be enrolled at a 500mg dose (Part 1), followed by a further 12 (if efficacy still shown) at a dose of 250mg (Part 3) to determine the minimum effective dose of BCX7353 compared to placebo for treating HAE attacks. Efficacy will be determined by subject diary entries completed at pre-defined times post-dose.

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Key information

Conditions

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

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Study Center, Graz, Austria

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Able to provide written, informed consent.
A clinical diagnosis of hereditary angioedema Type 1 or Type 2 as documented at any time in the medical records or at the screening visit.
Access to and ability to use standard of care acute attack treatment for attacks of HAE.
Sexually active women of child-bearing potential and sexually active men must utilize effective contraception.

Exclusion criteria

Women who are pregnant or breast-feeding.
Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study.
Use of C1INH, androgens or tranexamic acid for prophylaxis of HAE attacks.
History of or current alcohol or drug abuse.
Infection with hepatitis B, hepatitis C or HIV.
Participation in any other investigational drug study currently or within the last 30 days.
Positive drugs of abuse screen (unless as used as medical treatment, e.g., with a prescription).
An immediate family relationship to either Sponsor employees, the Investigator or employees of the study site.

Treatment and study plan

BCX7353

Drug

oral liquid formulation

Placebo

Drug

oral liquid formulation to match BCX7353

Primary outcomes

Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score

Time frame: Mean composite VAS for HAE attack symptoms severity prior to IMP treatment and 4 hours post-dose

Subjects completed a 3-component VAS on a 100 mm scale for severity of abdominal pain, skin pain and skin swelling associated with the HAE attack, where zero indicated no pain or swelling and 100 mm indicated worst possible pain or swelling. Subjects completed the VAS immediately prior to study drug administration, then at 1, 2, 3, 4, approximately 8 & at 24 hours post-dose. The primary endpoint was the proportion of subject attacks with an improved or stable 3-symptom composite VAS score at 4 hours post dose. The 3-symptom composite was calculated as the average of the VAS scores for abdominal pain, skin pain, and skin swelling. A subject was considered improved or stable if the change from baseline (CFB; time of drug administration) in VAS was ≤ 0.
Percentage of Attacks Treated With Standard of Care Acute Attack Medication (SOC-Rx) Through 24 Hours

Time frame: 24 hours

The proportion of attacks for which subjects took SOC-Rx in the 24 hours following treatment with study drug. HAE Rescue Medications included C1-INH (Berinert, Cinryze, Ruconest) and Firazyr/Icatibant.

Sponsors and collaborators

Lead sponsor

BioCryst Pharmaceuticals

Industry

Registry information

Official study title

A Randomized, Double-blind, Placebo-controlled, Dose-ranging, Study to Evaluate the Efficacy, Safety and Tolerability of Single Doses of BCX7353 as an Acute Attack Treatment in Subjects With Hereditary Angioedema

Important dates

Study start: 2017
Primary completion: 2019
Study completion: 2019
First posted: Aug 4, 2017
Registry last updated: Apr 1, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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Completed