Hereditary Angioedema MedDRA version: 18.0 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: avoralstat
Product Code: BCX4161
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: avoralstat
CAS Number: 918407-35-9
Other descriptive name: BCX4161
Concentration unit: mg milligr
Sponsors
BioCryst Pharmaceuticals Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Males and non-pregnant, non-lactating females age = 18 years 2) Prior successful completion of an avoralstat therapeutic study OR Laboratory confirmed diagnosis of HAE Type 1 (low C1INH antigenic level) or HAE Type 2 (low C1 INH functional level) 3) Agree by the Investigator and subject that the subject is a suitable candidate for prophylaxis of HAE attacks 4) Access to Appropriate medication for the treatment of acute HAE attacks 5) Female subjects must meet one of the following requirements: a) A woman of childbearing potential (defined as a non-menopausal female who has not had a hysterectomy, bilateral oophorectomy, or documented ovarian failure) who agrees to use a highly effective method of contraception for the study duration and for 30 days after study drug dosing. The following methods are considered to be highly effective methods of contraception: i) surgical sterilization (i.e., bilateral tubal ligation) ii) partner’s surgical sterilization (i.e., vasectomy) iii) placement of an intrauterine device (IUD) or intrauterine system iv) hormonal contraception (implantable, patch, oral, vaginal ring) v) 2 barrier methods (their partner’s use of a condom and the subject’s use of an occlusive cap [diaphragm, or cervical/vault caps] with spermicidal foam/gel/film/cream/suppository) Female subjects who report being postmenopausal for = 2 years and have a screening follicle stimulating hormone (FSH) = 40 mIU/mL must agree to use a highly effective contraceptive method outlined above for the study period and for 30 days after the last dose of study drug. b) A woman of non-childbearing potential (defined as being postmenopausal for > 2 years, having a screening FSH > 40 mIU/mL if postmenopausal for = 2 years, or a woman who has had a hysterectomy, bilateral oophorectomy, or documented ovarian failure) c) A woman declaring herself as either sexually abstinent or exclusively having female sexual partners. Abstinence in this study is defined as "True abstinence: when this is in line with the preferred and usual lifestyle of the subject." All women of childbearing potential making this declaration must consent to utilizing a highly effective method of birth control prior to engaging in any sexual activity with male partners through 30 days after the last dose of study drug. 6) Male subjects must comply with the following requirements a) Sexually active male subjects must agree to utilize one highly effective contraceptive method with female partners of childbearing potential (defined as postmenopausal = 2 years or a non-menopausal female who has not had a hysterectomy, bilateral oophorectomy, or documented ovarian failure) through the study period and for 90 days after the last dose of study drug. A “highly effective contraceptive method” is defined as one of the following: i) subject having had a vasectomy ii) partner’s surgical sterilization (i.e., bilateral tubal ligation) iii) partner’s placement of an intrauterine device or intrauterine system iv) partner’s use of hormonal contraception (implantable, patch, oral, vaginal ring) v) 2 barrier methods (their partner’s use of an occlusive cap [diaphragm, or cervical/vault caps] with spermicidal foam / gel / film / cream / suppository and the subject’s use of a condom) b) Male subjects who declare themselves as sexually abstinent are acceptable for the purposes of this study. Abstinence in this study is defined as “true abstinence: when this is in line with the preferred and usual l
Exclusion criteria
Exclusion criteria: 1) Females who are pregnant (positive urine or serum pregnancy) or breast feeding at the screening visit or who are planning to become pregnant during the study period 2) Any clinically significant medical condition or medical history that, in the opinion of the Investigator or Sponsor, would interfere with the subject’s safety or ability to participate in the study 3) Any significant comorbid condition that is not controlled (e.g., gastrointestinal disease that leads to malabsorption) 4) Dementia, altered mental status or any psychiatric condition, or stay in an institution further to an official or court order that would prohibit the understanding or rendering of informed consent or participation in the study 5) Current or past medical history of chronic pancreatitis, pancreatic insufficiency at any time in the past, or a confirmed history of acute pancreatitis occurring within the past 5 years 6) Clinically significant abnormal ECG as assessed by the Investigator 7) Any abnormal laboratory or urinalysis finding at the screening visit that, in the opinion of the Investigator or Sponsor, is clinically significant and relevant for this study 8) Investigational drug exposure in any other investigational drug study within 30 days prior to the screening visit (with the exception of an investigational study of avoralstat) 9) History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse (self-reported alcoholic intake > 3 units alcohol/day) 10) For subjects undergoing a screening visit, a positive drugs of abuse screen (unless as used as medical treatment, e.g., with a prescription) 11) For subjects undergoing a screening visit, infection with hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the long-term safety and tolerability of oral avoralstat in subjects with hereditary angioedema (HAE) ;Secondary Objective: To assess HAE attack frequency, severity and disease activity To evaluate quality of life ;Primary end point(s): Safety endpoints will include the proportion of subjects: who discontinue due to a treatment-emergent AE; with treatment-emergent serious adverse event (SAE); with treatment emergent Grade 3 or 4 AE; with treatment emergent Grade 3 or 4 laboratory abnormalities.;Timepoint(s) of evaluation of this end point: At least annually, and when the last subject discontinues treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The efficacy endpoints will include acute angioedema attack rate, durability of response, number of attack free days, acute HAE attacks requiring medication, discontinuations due to lack of efficacy, and quality of life over time.;Timepoint(s) of evaluation of this end point: At least annually, and when the last subject discontinues treatment | — |
Countries
Belgium, Canada, France, Germany, Hungary, Italy, United Kingdom, United States
Contacts
Public ContactClinical Operations
AMS Advanced Medical Servicess
Outcome results
None listed