Hereditary Angioedema (HAE) MedDRA version: 23.1 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
BioCryst Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and non-pregnant, non-lactating females 2 to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Concurrent diagnosis of any other type of recurrent angioedema. 2. Any clinically significant history of angina, myocardial infarction, syncope, clinically significant cardiac arrhythmias, left ventricular hypertrophy, cardiomyopathy, myocarditis, pericarditis, congenital heart defects, or any other clinically significant cardiovascular abnormality such as poorly controlled hypertension. 3. Known family history of sudden cardiac death at a young age (ie, 450 msec, or ventricular and/or atrial premature contractions that are more frequent than occasional, and/or as couplets or higher in grouping. 13. Known hypersensitivity to berotralstat or any of its formulation excipients.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To describe the pharmacokinetic (PK) parameters of berotralstat administered orally to pediatric subjects with HAE aged 2 to < 12 years old and weighing = 12 kg.;Secondary Objective: - To assess the safety and tolerability of berotralstat administered orally to pediatric subjects with HAE aged 2 to < 12 years old and weighing = 12 kg. - To summarize the effectiveness of berotralstat in pediatric subjects with HAE aged 2 to < 12 years old and weighing = 12 kg.;Primary end point(s): PK : The primary endpoint is the characterization of the PK profile of berotralstat in subjects aged 2 to < 12 years.;Timepoint(s) of evaluation of this end point: The primary endpoint will be evaluated at week 2, part 1 and at week 48, part 2 of the study for each cohort of patients. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Safety : The frequency and severity of adverse events (AEs) and serious adverse events (SAEs), laboratory analyses (clinical chemistry, hematology, coagulation), height, weight, vital signs, electrocardiograms (ECGs), and physical examination findings Effectiveness: The frequency of attacks, duration of symptoms, anatomical location of attack, on-demand treatment, number of days with angioedema symptoms, assessment of attack severity, discontinuations due to lack of efficacy, and number of hospitalizations and clinic visits from Week 1 through Weeks 12 and 48;Timepoint(s) of evaluation of this end point: Safety endpoints will be assessed at the end of each Part (ie, Weeks 12, 48, and 144/end of study [EOS]). The other secondary endpoint of effectiveness will be measured by assessing the frequency of attacks, duration of symptoms, anatomical location, on-demand treatment required, number of days with angioedema symptoms, assessment of attack severity, discontinuations due to lack of efficacy, and number of hospitalizations and clinic visits from Week 1 through Weeks 12 and 48. | — |
Countries
Austria, Canada, France, Germany, Israel, Italy, Poland, Romania, Spain, United Kingdom
Contacts
Public ContactChristine Cordes
Syneos Health
Outcome results
None listed