BCX7353 in prevention of HAE attacks
Research type
Research Study
Full title
A randomized, double-blind, 4 week, placebo-controlled, dose-ranging, parallel-group study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of BCX7353 as a preventative treatment to reduce the frequency of attacks in subjects with hereditary angioedema
IRAS ID
193120
Contact name
Angela Rose
Contact email
Sponsor organisation
BioCryst Pharmaceuticals Inc
Eudract number
2015-003923-74
Clinicaltrials.gov Identifier
5238, CRN
Duration of Study in the UK
0 years, 5 months, 27 days
Research summary
Hereditary angioedema (HAE) is an autosomal dominant disorder characterized by periodic episodes of swelling of the skin, pharynx, larynx, gastrointestinal tract, genitals, and extremities. The frequency of these attacks varies between\nsubjects, with attacks occurring rarely in some patients to as frequently as every few days in others. HAE attacks, which may or may not be precipitated by a stimulus (such as stress, trauma, or estrogen) are typically slow in onset, peaking in intensity within 24 hours, and gradually subside over the subsequent 5 days. This study will assess the ability of a novel orally administered compound BCX7353 to prevent these attacks. The study assesses three different doses of BCX7353 compared to placebo over a 28 days treatment period. Patients will complete electronic diaries to document the attacks they have, and how they treat them. Patients will be allowed to treat their attacks as per normal clinical practice, without restriction. Subject reported attacks will be adjudicated by an independent committee and the primary analysis will be of active versus placebo weekly attack frequencies.
REC name
London - Chelsea Research Ethics Committee
REC reference
15/LO/2014
Date of REC Opinion
23 Dec 2015
REC opinion
Favourable Opinion