BCX7353 in prevention of HAE attacks
Research type
Research Study
Full title
A randomized, double-blind, placebo-controlled, dose-ranging, parallel-group study to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of BCX7353 as a preventative treatment to reduce the frequency of attacks in subjects with hereditary angioedema
IRAS ID
205417
Contact name
Melanie Cornpropst
Contact email
Sponsor organisation
BioCryst Pharmaceuticals Inc
Eudract number
2016-001272-29
Duration of Study in the UK
2 years, 1 months, 12 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
subjects, 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.
Initially a dose of 350mg once a day will be compared to placebo in upto 36 patients. If this is shown to be effective, then upto a further 54 patients will be enrolled to try and determine the minimum effective dose of BCX7353 compared to placebo for preventing HAE attacks.
Patients will complete 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 attack frequenciesREC name
London - London Bridge Research Ethics Committee
REC reference
16/LO/0858
Date of REC Opinion
26 Jul 2016
REC opinion
Further Information Favourable Opinion