Hereditary Angioedema
Conditions
Interventions
Trade Name: Firazyr® 30 mg subcutaneous injection Syringe: 3.0 mL×1 Syringe
Product Name: Icatibant Acetate (JAN)
Product Code: TAK-667
Pharmaceutical Form: Injection
INN or Proposed INN: Icatibant
CA
Sponsors
Takeda Pharmaceutical Company Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. In the opinion of the investigator or subinvestigator, the subject’s parent or legal guardian is capable of understanding and complying with protocol requirements. 2. The subject’s parent or the subject’s legal guardian is capable of signing and dating a written informed consent form on behalf of the subject prior to the initiation of any study procedures. Written informed assent is also obtained from the subject as much as possible. 3. The subject is in Japan and is Japanese; defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents. 4. The subject is male or female and 2 to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. The subject will require an intervention to support the airway (eg, intubation, tracheotomy, cricothyrotomy) due to the current HAE attack. 2. The subject 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 subject has a diagnosis of angioedema other than HAE 4. The subject 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 subject has received treatment with any pain medication since the onset of the current HAE attack. 6. The subject 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 subject has received treatment with ACE inhibitors within 7 days prior to treatment. 8. The subject has used hormonal contraceptive within 90 days prior to treatment. 9. The subject has received androgen or attenuated androgens (eg, danazol, testosterone) within 90 days prior to treatment. 10. The subject has participated in another clinical study within the past 30 days before screening. 11. The subject, the subject’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 subject 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 subject has a history of hypersensitivity or allergies to icatibant. 14. The subject is judged by the investigator as being ineligible for any other reason; eg. a serious concomitant illness or condition.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety, efficacy and PK of icatibant for the treatment of acute attacks in Japanese children and adolescents with type I or type II HAE.;Secondary Objective: Not applicable;Primary end point(s): Frequency and severity of treatment-emergent adverse events (TEAEs), including injection site reactions;Timepoint(s) of evaluation of this end point: Primary end point will be assessed throughout the study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Resting 12-lead electrocardiogram parameters in comparison to baseline - Vital sign measurements in comparison to baseline - Clinical laboratory parameters (serum chemistry, hematology and urinalysis) in comparison to baseline - Reproductive hormone levels in comparison to baseline - Immunogenicity (presence of anti-icatibant antibodies) in comparison to baseline - Time to onset of symptom relief and time to minimal symptom, as measured by investigatorand subject-reported outcomes. a) For all subjects (2 to <18 years of age): investigator assessment and scoring of cutaneous, abdominal, and laryngeal symptoms of acute HAE attacks by an investigator-rated symptom score. 1. The time to onset of symptom relief 2. The time to minimal symptoms b) For subjects =4 to <18 years of age only: subject self-assessment of HAE-related pain using the Faces Pain Scale-Revised (FPS-R). 1. The time to onset of symptom relief 2. The time to minimal symptoms c) For subjects 2 to <4 years of age only: investigator assessment of HAE-related pain using the Faces, Legs, Activity, Cry, and Consolability (FLACC) scale. 1. The time to onset of symptom relief 2. The time to minimal symptoms - The incidence of rescue medication use - The proportion of subjects with worsened intensity of clinical HAE symptoms between 2 and 4 hours after treatment with SC icatibant using investigator-rated symptom scores - Time to initial symptom improvement reported by investigator or subject Time to initial symptom improvement reported by subject - Plasma concentrations of icatibant and its major metabolites (M-I and M-II) at 0.5, 1.0, 2.0, and 4.0 hours after the first SC injection for an initial attack;Timepoint(s) of evaluation of this end point: Secondary end points will be assessed throughout the study. | — |
Countries
Japan
Contacts
Public ContactStudy Director
Takeda Pharmaceutical Company Limited
Outcome results
None listed