JPRN-jRCT2041200073已完成3 期
A Multicenter, Open-Label, Non-randomized Phase 3 Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneous Administration of Icatibant (TAK-667) in Japanese Children and Adolescents with Acute Attacks of Hereditary Angioedema
Koumura Emiko0 个研究点目标入组 2 人开始时间: 2020年12月15日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 2
研究概览
简要总结
In conclusion, this study demonstrated that icatibant can provide a safe and effective treatment for the unmet medical need of acute HAE attacks in Japan pediatric patients.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 2age old 至 < 18age old（—）
- 性别
- All
入选标准
- •1. In the opinion of the investigator or subinvestigator, the participant's parent or legal guardian is capable of understanding and complying with protocol requirements.
- •2. The participant's parent or the participant's legal guardian is capable of signing and dating a written informed consent form on behalf of the participant prior to the initiation of any study procedures. Written informed assent is also obtained from the participant as much as possible.
- •3. The participant is in Japan and is Japanese; defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents.
- •4. The participant is male or female and 2 to <18 years of age (ie, from the second birthday through the day prior to the eighteenth birthday) at the time of informed consent.
- •5. The participant weighs >=12 kg at the time of the current HAE attack.
- •6. The participant who has a documented and confirmed diagnosis of HAE type I or II. Diagnosis
- •may be based on historical data using the following criteria:
- •a. Family history of angioedema
- •b. Characteristic attack manifestations, recurrent attacks
- •c. Functional complement 1 (C1) esterase inhibitor (C1-INH) deficiency
- •d. In the absence of a family history of angioedema, exclusion of other forms of angioedema (eg. angiotensin converting enzyme (ACE)-induced angioedema, allergic angioedema) based on medical judgement (eg, concomitant medication, response to antihistamines or glucocorticoids, information of genetic mutation).
- •7. If the participant does not have a documented and confirmed diagnosis of HAE type I or II based on historical data, including C1-INH deficiency, the participant's diagnosis must be determined prior to treatment by C1-INH test results which demonstrate a functional C1-INH deficiency.
- •a. HAE type I: Low amount of C1-INH protein and low level of C1-INH activity; HAE type II: Normal or increased amount of C1-INH protein and low level of C1-INH activity
- •b. In the absence of a family history of angioedema, exclusion of other forms of angioedema (eg. ACE-induced angioedema, allergic angioedema) based on medical judgement (eg, concomitant medication, response to antihistamines or glucocorticoids, information of genetic mutation).
- •8. The current HAE attack must be in the cutaneous, abdominal, and/or laryngeal (inclusive of laryngeal and pharyngeal) areas, but no prespecified attack severity criteria are required for
- •9. The participant commences treatment within 12 hours after the onset of current HAE attack.
- •10. A female participant of childbearing potential who is sexually active with a nonsterilized male partner agrees to use routinely adequate contraception from signing of informed consent throughout the duration of the study, and proves negative in the pregnancy test at screening.
排除标准
- •1. The participant will require an intervention to support the airway (eg, intubation, tracheotomy, cricothyrotomy) due to the current HAE attack.
- •2. The participant presents with an HAE attack with laryngeal/upper respiratory tract symptoms which are considered severe in the investigator's clinical judgment and which may necessitate urgent care and/or impede the conduct of study efficacy assessments.
- •3. The participant has a diagnosis of angioedema other than HAE
- •4. The participant has evidence of stroke or coronary artery disease based on medical history at the screening examination or at pretreatment; eg, acute ischemic heart disease, unstable angina pectoris, severe coronary heart disease or congestive heart failure, that in the investigator's judgment would be a contraindication for participation in the trial (New York Heart Association [NYHA] class 3 and 4).
- •5. The participant has received treatment with any pain medication since the onset of the current HAE attack.
- •6. The participant has received replacement therapy (C1-INH products, fresh frozen plasma [FFP]) within 5 days (120 hours) from the onset of the current HAE attack.
- •7. The participant has received treatment with ACE inhibitors within 7 days prior to treatment.
- •8. The participant has used hormonal contraceptive within 90 days prior to treatment.
- •9. The participant has received androgen or attenuated androgens (eg, danazol, testosterone) within 90 days prior to treatment.
- •10. The participant has participated in another clinical study within the past 30 days before screening.
- •11. The participant, the participant's parent, or legal guardian is unable to understand the nature, scope, and possible consequences of the protocol, or is unlikely to comply with the protocol assessments, unable to return for follow up visits, or unlikely to complete the study for any reason.
- •12. If female, the participant is pregnant or lactating or intending to become pregnant before participating in this study, during the study, and within 30 days after last dose of the icatibant.
- •13. The participant has a history of hypersensitivity or allergies to icatibant.
- •14. The participant is judged by the investigator as being ineligible for any other reason; eg. a serious concomitant illness or condition.
研究者
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