BCX7353 as a treatment for acute HAE attacks
Research type
Research Study
Full 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
IRAS ID
207238
Contact name
Melanie Cornpropst
Contact email
Sponsor organisation
BioCryst Pharmaceuticals Inc
Eudract number
2016-001424-55
Duration of Study in the UK
1 years, 6 months, 16 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 treat these attacks.
Each patient will treat three attacks, two with active, and one with placebo in a blinded randomised manner. Initially a dose of 750mg will be assessed against placebo. If efficacious, then the study proceeds to Part 2 assessing 500mg, followed by 250mg if 500mg is shown to work.
It is anticipated that approximately one attack per month would be treated by the patients.REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
17/EE/0204
Date of REC Opinion
20 Jun 2017
REC opinion
Further Information Favourable Opinion