NL-OMON48865已完成2 期
An Open-Label study to Evaluate the Long-Term Safety of Daily Oral BCX7353 in subjects with Type I and II Hereditary Angioedema - A long term study of BCX7353 in the prevention of HAE attacks
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 3
研究概览
简要总结
Trial is onging in other countries
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •1. Males and nonpregnant, nonlactating females >=18 years of age (main study) ;
- •2. Subjects with Type I or II HAE who have either participated in a BCX7353
- •efficacy study OR
- •expected to derive benefit from oral treatment for the prevention of angioedema
- •attacks and have a clinical diagnosis of HAE Type 1 or Type2, defined as C1-INH
- •functional level below 50% and C4 level below lower limit of normal OR a known
- •SERPING-1 gene mutation known or likely to be associated with HAE Type 1 or 2
- •OR confirmed family history OR low C4 value from a sample drawn during a HAE
- •3. Subject weight >= 40kg
- •4. Access to appropriate medication for the treatment of acute HAE attacks
- •5. Females only - Agreement to use acceptable effective contraception
- •6. Able to provide written, informed consent
- •7. In the opinion of the Investigator, the subject is able to adequately comply
- •with all required study procedures for the duration of the study. The subject
- •must demonstrate adequate compliance with all study procedures required
- •including diary recording of HAE attacks
排除标准
- •1. Pregnancy or breast feeding or planned pregnancy 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. Discontinuation of study drug due to a hypersensitivity reaction to BCX7353
- •in a prior study. This includes subjects who had a rash of any severity
- •identified as possibly, probably, or definitely related to active BCX7353 in
- •the previous study
- •4. Dementia, altered mental status or any psychiatric condition, that would
- •prohibit the understanding or rendering of informed consent or participation in
- •5. Clinically significant abnormal ECG including but not limited to, a QTcF >
- •470 msec for women, a QTcF > 450 msec for men, a PR > 220 msec (both sexes), or
- •ventricular and/or atrial premature contractions that are more frequent than
- •occasional, and/or as couplets or higher in grouping
- •6. Unacceptable noncompliance in the previous BCX7353 efficacy study as
- •assessed by the Sponsor or Investigator
- •7. Any clinically significant history of angina, myocardial infarction,
- •syncope, clinically significant cardiac arrhythmias, left ventricular
- •hypertrophy, cardiomyopathy, or any other cardiovascular disease
- •8. Known family history of sudden cardiac death. Family history of sudden death
- •from HAE is not exclusionary
- •9. History of or current implanted defibrillator or pacemaker
- •10. Use of concomitant medications that are metabolized by CYP2D6, CYP2C9,
- •CYP2C19, or CYP3A4 and have a narrow therapeutic range, within 7 days of the
- •baseline visit or planned initiation during the study, including those known to
- •prolong the QT interval
- •11. Use of a medication that is transported by P-gp and has a narrow
- •therapeutic range, within 7 days of the baseline visit or planned initiation
- •during the study
- •12. Any laboratory parameter abnormality that, in the opinion of the
- •Investigator, is clinically significant and relevant for this study
- •13. Calculated creatinine clearance (CLcr) of <= 60 mL/min or AST or ALT value >=
- •2 times the ULN reference range value at screening or at the last available
- •visit prior to enrollment
- •14. Investigational drug exposure, other than BCX7353, within 30 days prior to
- •the screening visit (or baseline if no screening visit)
- •15. History of severe hypersensitivity to any medicinal product, which was
- •associated with non HAE-related swelling, a severe rash requiring treatment /
- •hospitalization, or anaphylaxis
- •16. History of alcohol or drug abuse within the previous year, or current
- •evidence of substance dependence or abuse (self-reported alcoholic intake > 3
- •units of alcohol/day)
- •17. For subjects undergoing a screening visit, a positive drugs of abuse screen
- •(unless used as a medical treatment [e.g., with a prescription])
- •18. For subjects undergoing a screening visit, current infection with hepatitis
- •B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)
- •19. Subjects with an immediate family relationship to either Sponsor employees,
- •the Investigator or employees of the study site who are named on the delegation
- •20. Subjects who are held in an institution by a government or judicial order
研究者
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