A Study of Icatibant (TAK-667) in Japanese Children and Teenagers With Acute Attacks of Hereditary Angioedema

What Was Tested

The trial (phase 3) is built around Icatibant.

The main aim of the study is to check for side effects from icatibant in children and teenagers with hereditary angioedema (HAE).

Other aims are to check how well icatibant controls HAE symptoms in these children and teenagers, and how much icatibant stays in their blood.

At the first visit, the study doctor will check if each child or teenager can take part.

It had 1 arm (groups): TAK-667 (experimental).

The registry classes the interventions as drug.

More on the Plan

The drug being tested in this study is called TAK-667.

TAK-667 is being tested to treat Japanese Children and Adolescents who experience acute attack of HAE.

The study will enroll at least up to 3 participants.

Participants will take TAK-667 with SC administration on the abdomen.

Who Took Part

2 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: In the opinion of the investigator or subinvestigator, the participant's parent or legal guardian is capable of understanding and complying with protocol requirements. The participant is in Japan and is Japanese; defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents. The participant weighs \>=12 kg at the time of the current HAE attack. 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:. Family history of angioedema.

The full record lists 16 inclusion and 14 exclusion criteria; among the exclusions: the participant will require an intervention to support the airway (eg, intubation, tracheotomy, cricothyrotomy) due to the current hae attack; the participant has a diagnosis of angioedema other than hae; the participant has received treatment with any pain medication since the onset of the current hae attack.

How the Study Was Run

It was run as a single-group and open-label study.

Everyone knew when Icatibant was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

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

It started in 2021-01-15 with completion listed for 2021-07-27.

It ran at 3 sites in Japan (3).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Number of Participants Who Experienced at Least One Treatment-Emergent Adverse Events (TEAE) (time frame: Up to approximately 6 months). Number of Participants With Injection Site Reactions (time frame: Postdose, up to Day 8).

It also tracked 15 secondary outcomes, for example Number of Participants Who Experienced at Least One TEAE Related to Resting 12-lead Electrocardiogram, Number of Participants Who Experienced at Least One TEAE Related to Vital Sign, Number of Participants Who Experienced at Least One TEAE Related to Clinical Laboratory Parameters, Number of Participants Who Reported Presence of Anti-icatibant Antibodies, Time to Onset of Symptom Relief With Investigator-Rated Symptom Scores Assessed by Investigator and Time to Onset of Symptom Relief With Faces Pain Scale-Revised (FPS-R) Scores for Participants of 4 Years Age and Older.

What This Means in Plain Language

Put simply, this is a phase 3 trial of Icatibant in 2 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.