This trial is active, not recruiting.

Condition hereditary angioedema
Treatment icatibant
Phase phase 3
Sponsor Shire
Start date December 2011
End date December 2016
Trial size 30 participants
Trial identifier NCT01386658, 2011-003825-81, HGT-FIR-086


HGT-FIR-086 is a multicenter, open-label, non-randomized, single-arm study to evaluate the Pharmacokinetics, tolerability, and safety including effect on reproductive hormones, of a single subcutaneous, (SC) administration of icatibant in approximately 30 pediatric subjects with Hereditary Angioedema (HAE) during an initial acute attack.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification pharmacokinetics study
Intervention model single group assignment
Masking open label
Primary purpose treatment
icatibant Firazyr
Single dose of icatibant 0.4 mg/kg subcutaneous(SC) up to a maximal dose of 30 mg

Primary Outcomes

Pharmacokinetic (PK) Profile after a single SC injection (in prepubertal children with an acute attack of HAE and pubertal/postpubertal children with or without an acute attack of HAE)
time frame: Administration through 6 hours
Safety of a single SC dose of icatibant
time frame: Treatment through day 90

Secondary Outcomes

Time to onset of relief of symptoms and time to minimal symptoms, as measured by investigator- and subject-reported outcomes for subjects who have experienced HAE attack only
time frame: Treatment through 8 hours
Proportion of subjects with worsened intensity of clinical HAE symptoms between 2 and 4 hours after treatment with icatibant for subjects who have experienced HAE attack only
time frame: Treatment through 4 hours
Incidence of rescue medication use for subjects who have experienced HAE attack only
time frame: Treatment through day 90

Eligibility Criteria

Male or female participants from 2 years up to 17 years old.

Inclusion Criteria: 1. Two through <18 years of age at the time of first HAE attack. - Prepubertal and pubertal/postpubertal subjects experiencing an HAE attack. - Pubertal/postpubertal subjects with HAE who are treated with icatibant, but not during an attack. 2. Documented diagnosis of HAE Type I or II. 3. Informed consent (and subject assent as appropriate) signed by the subject's parent(s)or legal guardian(s). Exclusion Criteria: 1. Diagnosis of angioedema other than HAE. 2. Participation in another clinical trial that involves the use of any investigational product (drug or device)within 30 days prior to study enrollment or at any time during the study. 3. Any known factor/disease that might interfere with the treatment compliance, study conduct,or result interpretation. 4. Congenital or acquired cardiac anomalies that interfere significantly with cardiac function. 5. Treatment with ACE inhibitors within 7 days prior to treatment. 6. Use of hormonal contraception within the 90 days prior to treatment. 7. Androgen use (eg, stanozolol, danazol, oxandrolone, methyltestosterone, testosterone) within the 90 days prior to treatment. 8. Pregnancy or breastfeeding. 9. A physical condition that interferes with pubertal status determination.

Additional Information

Official title A Multicenter, Open-Label, Non-Randomized Study to Assess the Pharmacokinetics, Tolerability, and Safety of a Single Subcutaneous Administration of Icatibant in Children and Adolescents With Hereditary Angioedema
Description Study HGT-FIR-086 will enroll 30 subjects from 2 to less than 18 years of age, divided into 2 groups: prepubertal and pubertal/postpubertal. At least 10 prepubertal children and at least 20 adolescents (including 10 treated during a HAE attack) must be enrolled in the study. After a qualifying screening period, the PK, safety/tolerability, and efficacy of treatment with SC icatibant will be evaluated in at least 20 subjects (10 prepubertal and 10 pubertal/postpubertal subjects) who present with cutaneous, abdominal, or laryngeal symptoms of an acute attack of HAE. The PK and safety/tolerability of SC icatibant will be evaluated in at least 10 additional pubertal/postpubertal subjects who meet screening criteria and receive treatment with SC icatibant in the absence of a current acute HAE attack. The planned duration of active participation for subjects who present with an initial attack of acute HAE will consist of treatment with a single subcutaneous injection of icatibant on Day 1 through follow up at day 90. After having received initial treatment with icatibant, either during or in the absence of an attack, at least 10 pubertal/postpubertal subjects who subsequently experience an acute HAE attack may continue to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant-treated attacks. The period of active participation in the study for prepubertal subjects will be approximately 90 days, while that for pubertal/postpubertal subjects could be a maximum of approximately 270 or 360 days (3 separate active periods of approximately 90 days for those treated with icatibant during an attack; 4 separate active periods for those treated without an attack), with each active period separated by periods of inactive participation of variable duration.
Trial information was received from ClinicalTrials.gov and was last updated in November 2015.
Information provided to ClinicalTrials.gov by Shire.