EUCTR2021-005932-50-AT进行中（未招募）1 期
A PHASE 3 STUDY TO EVALUATE THE SAFETY AND PHARMACOKINETICS OF BEROTRALSTAT PROPHYLAXIS IN CHILDREN WITH HEREDITARY ANGIOEDEMA WHO ARE 2 TO < 12 YEARS OF AGE
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 30
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Male and non-pregnant, non-lactating females 2 to < 12 years of age and weighing = 12 kg.
- •2. Parent/caregiver willing and able to provide written, informed consent (with assent from the child where appropriate).
- •3. Subjects with a clinical diagnosis of HAE. A clinical diagnosis of HAE is defined as:
- •a. Screening results that document immunogenic C1 esterase inhibitor (C1-INH) antigenic level below the lower limit of normal (LLN) reference range or C1-INH function < 50%, and a complement 4 (C4) level below the LLN reference range.
- •b. Laboratory documentation of historical C1-INH functional level below the assay lower limit of normal.
- •c. For subjects with C1-INH function = 50% but less than the assay LLN, a SERPING-1 gene mutation known or likely to be associated with HAE Type I or II, as assessed during the screening period OR a repeat C1-INH functional level < 50% will be considered acceptable for enrollment.
- •d. Historical or new laboratory documentation of a SERPING-1 mutation known or likely to be associated with HAE.
- •e. For subjects who currently use plasma derived or recombinant C1-INH based prophylactic therapies, a confirmed family history of C1-INH deficiency.
- •4. For subjects who are not currently receiving prophylaxis for HAE, documented history of = 2 HAE attacks in the 6 months prior to the enrollment visit.
- •5. Access to and ability to use one or more acute medications approved by the relevant competent authority for the treatment of acute attacks of HAE.
- •6. In the opinion of the investigator, the subject would benefit from long-term oral prophylaxis.
- •7. Females who had started their menses and males must either:
- •a. Be sexually abstinent. Abstinence in this study is defined as true abstinence: when this is in line with the preferred and usual lifestyle of
- •the subject.
- •b. Use contraception. The following represents the minimum contraception that should be used by subjects who could be sexually active. Additional contraceptive requirements, such as highly effective methods, may be required by local site practice and/or governing institutional review board/ethics committee. It is anticipated that not all contraceptive methods may be available in all countries/regions, so the list should be modified accordingly.
- •Male subjects should use condoms while enrolled in the study.
- •Female subjects should use one or more of the following methods during the study and 30 days after the last dose of berotralstat:
- •- Intrauterine device (IUD) or intrauterine hormone releasing system (IUS)
- •- Progesterone-only (implantable or injectable only) or oral (norethindrone based only) hormonal contraception
- •- Combined (estrogen and progestogen containing) oral, intravaginal, or transdermal hormonal contraception associated with inhibition of ovulation
- •Note: Angioedema attacks may be precipitated by estrogen.
- •- Male or female condom with or without spermicide
- •- Use of an occlusive cap (diaphragm, or cervical/vault caps) with spermicide (foam/gel/film/cream/suppository)
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 30
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •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, < 40 years of age). Family history of sudden death from HAE is not exclusionary.
- •4. History of or current implanted defibrillator or pacemaker.
- •5. Moderate to severe hepatic impairment (Child-Pugh B or C).
- •6. A calculated creatinine clearance using the Modified Schwartz formula of = 30 mL/min/1.73 m2 or aspartate aminotransferase or alanine aminotransferase value = 3 × the upper limit of the age-appropriate normal reference range value.
- •7. History of severe hypersensitivity to multiple medicinal products or severe hypersensitivity/anaphylaxis with unclear etiology.
- •8. Current participation in any other investigational drug study or received another investigational drug within 30 days of enrollment; not willing to refrain from participation in another clinical study after enrollment and for the duration of the study. [Note: drugs/vaccines approved under FDA emergency use authorization (or country-specific analogous regulations) are not considered excluded or prohibited under this criterion.]
- •9. An immediate family relationship to either sponsor employees, the investigator, or employees of the study site named on the delegation log.
- •10. Any result at screening that, in the opinion of the investigator, is clinically significant and relevant for this study.
- •11. Any clinically significant medical condition or medical history (including altered mental status) that, in the opinion of the investigator or sponsor, would interfere with the subject's safety or ability to participate in the study. Examples include but are not limited to active malignancy under treatment, uncontrolled cardiovascular disease, organ dysfunction requiring supportive care.
- •12. Clinically significant abnormal ECG including but not limited to, a corrected QT interval calculated using Fridericia's correction (QTcF =QT/RR0.33) > 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.
研究者
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