OPuS 4 - Long term safety study of avoralstat
Research type
Research Study
Full title
OPuS-4: An open-label study to evaluate the long-term safety of avoralstat in subjects with hereditary angioedema
IRAS ID
189439
Contact name
Sylvia Dobo
Contact email
Sponsor organisation
BioCryst Pharmaceuticals Inc
Eudract number
2015-003242-22
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 safety of a novel compound avoralstat when used to prevent these attacks. Two previous studies OPuS1 and OPuS 2 assessed the efficacy of avoralstat. In OPuS 1, the 28 day proof of concept study of avoralstat in subjects with frequent HAE attacks, there was a reduction across all subjects of 0.45 attacks per week on avoralstat. OPuS 2 is ongoing, and results are not yet available, but the review of the safety data showed no new adverse effects on patients.
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.
Blood urine and faecal samples will be taken to assess safety of patients over long term exposure.REC name
London - Dulwich Research Ethics Committee
REC reference
15/LO/1678
Date of REC Opinion
13 Nov 2015
REC opinion
Favourable Opinion