A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema

What Was Tested

This registered phase 3 trial tested Icatibant.

It had 1 arm (groups): Icatibant (experimental).

The registry classes the interventions as drug.

More on the Plan

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.

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.

Who Took Part

The registry lists 32 actual participants took part.

It was open to people of any sex aged from 2 years up to 17 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Two through \<18 years of age at the time of first HAE attack. Prepubertal and pubertal/postpubertal subjects experiencing and acute cutaneous, abdominal, or laryngeal HAE attack treated with icatibant as part of this study. Pubertal/postpubertal subjects with HAE who are treated with icatibant, but not during an attack. Documented diagnosis of HAE Type I or II. Informed consent (and subject assent as appropriate) signed by the subject's parent(s)or legal guardian(s).

The full record lists 5 inclusion and 9 exclusion criteria; among the exclusions: diagnosis of angioedema other than hae; any known factor/disease that might interfere with the treatment compliance, study conduct,or result interpretation; treatment with ace inhibitors within 7 days prior to treatment.

How the Study Was Run

The set-up was single-group and open-label.

Nothing was hidden in this open-label Icatibant study: the participants and staff both knew the treatment.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2012-01-27 with completion listed for 2018-03-12.

It ran at 26 sites in United States (12), Germany (3), Israel (3), Spain (2), Australia (1) and Austria (1).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Number of Participants Who Reported Presence of Anti-icatibant Antibodies. Number of Participants With Adverse Events (AEs). Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 1.

It also tracked 13 secondary outcomes, for example Time to Onset of Symptom Relief (TOSR) for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 1, Time to Onset of Symptom Relief (TOSR) for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 2 and 3, Time to Onset of Symptom Relief (TOSR) for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 1, Time to Onset of Symptom Relief (TOSR) for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3, Time to Onset of Symptom Relief (TOSR) for Faces, Legs, Activity, Cry, and Consolability (FLACC) Scores and Time to Minimum Symptoms for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 1.

What This Means in Plain Language

Put simply, this is a phase 3 trial of Icatibant in 32 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Icatibant compound page, all human trials.