A Study of Icatibant (TAK-667) in Japanese Children and Teenagers With Acute Attacks of Hereditary Angioedema
- Conditions
- Hereditary Angioedema
- Registration Number
- JPRN-jRCT2041200073
- Lead Sponsor
- Koumura Emiko
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 2
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
treatment.
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.
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 1.Number of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE)<br>Time Frame: Up to approximately 6 months<br>An adverse event (AE) means any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. A treatment-emergent adverse event (TEAE) was defined as any adverse event occurring after the start of Icatibant administration of the treatment period.<br><br>2. Number of Participants with Injection Site Reactions<br>Time Frame: Post dose, up to Day 8<br>Injection sites were examined for erythema, swelling, cutaneous pain, burning sensation, itching/pruritus, and warm sensation.
- Secondary Outcome Measures
Name Time Method