Hereditary Angioedema 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. Males and non-pregnant, non-lactating females who are currently enrolled as a subject in BioCryst-sponsored Study 302 or 204 2. Able to provide written informed consent. Subjects who are aged 12 to 17 years of age must be able to read, understand, and be willing to sign an assent form in addition to an applicable representative providing informed consent. 3. Would benefit from continued berotralstat treatment in the opinion of the investigator 4. Female subjects must meet at least 1 of the following requirements: a. Be a WOCBP (defined as a female following menarche and prior to becoming post-menopausal who has not had a hysterectomy or bilateral salpingectomy and bilateral oophorectomy) who agrees to use at least an acceptable effective contraceptive method during the study. b. Be a woman of nonchildbearing potential (defined as postmenopausal [no menses for at least 12 months without an alternative medical cause] or has had a hysterectomy or a bilateral salpingectomy and bilateral oophorectomy). 5. In the opinion of the investigator, the subject is expected to adequately comply with all required study procedures. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 110 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Any condition or situation, including medical history or changes in medical history, which, in the opinion of the investigator or sponsor, would interfere with the subject’s safety or ability to participate in the study 2. Dementia, altered mental status, or any psychiatric condition that would prohibit the understanding or rendering of informed consent. 3. Hypersensitivity to the active substance or to any of the excipients in the IMP 4. Use of other medications for long-term prophylaxis of HAE attacks at the Baseline visit or any time during the study. These include: C1-INH, tranexamic acid, androgens, or lanadelumab. 5. Use of any other investigational medicinal product at the Baseline visit or any time during the study. 6. Pregnancy or breastfeeding. 7. Subjects with an immediate family relationship to either sponsor employees, the investigator, or employees of the study site who are named on the delegation log. 8. Subjects who are held in an institution by a government or judicial order.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To monitor the long-term safety of berotralstat;Secondary Objective: To provide continued access to berotralstat to subjects who have participated in Studies BCX7353-302 (Study 302) and BCX7353-204 (Study 204), are expected to continue benefiting from treatment with berotralstat, and do not have other means of access to berotralstat;Primary end point(s): Number and proportion of subjects with a treatment-related TEAE Number and proportion of subjects who experience a serious adverse event (SAE) Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality Number and proportion of subjects who discontinue due to a TEAE;Timepoint(s) of evaluation of this end point: Week 240 or until another mechanism is available to provide berotralstat (eg, market access). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Another mechanism is available to provide drug to the subject(eg, market access), or until the sponsor discontinues all global commercialization and development of berotralstat for the prevention of angioedema attacks, whichever comes first;Timepoint(s) of evaluation of this end point: Week 240 or until another mechanism is available to provide berotralstat (eg, market access). | — |
Countries
Czech Republic, European Union, France, Korea, Republic of, North Macedonia, Poland, Slovakia, South Africa
Contacts
Public ContactClinical Operations
AMS Advanced Medical Services
Outcome results
None listed