A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries
ConditionHereditary Angioedema (HAE)
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age2-11
SponsorTakeda
About this trial
HAE is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels but does not cause itching or hives. HAE is classified based on the amount of a protein in the blood called C1 inhibitor (C1-INH): HAE with normal C1-INH levels and HAE with limited or insufficient C1-INH levels (C1-INH deficiency). This study will concentrate on children with HAE C1-INH deficiency who have received Takhzyro (Lanadelumab) as prophylactic treatment.
The main goal of the study is to assess how well lanadelumab works in children with HAE-C1INH deficiency in everyday life. This will be measured by checking how long children who receive lanadelumab will be free of HAE attacks.
Other goals are to understand how children with HAE-C1INH deficiency are being treated with lanadelumab, how well the treatment works for them, how safe it is and how often these children need to use healthcare services (like doctor visits, hospital stays, etc.) because of their condition.
The study will only look at data already existing in the participants' medical records. No treatment will be given as part of the study.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Participant is aged 2 to less than (<) 12 years at the time of lanadelumab initiation and is expected to have at least 6 months of follow-up information before turning 12.
Participant has physician-confirmed diagnosis of HAE-C1INH.
Participant initiated LTP with lanadelumab during the eligibility period.
Signed consent/assent (where required by local regulations).
Disqualifiers
Participant was enrolled in a therapeutic investigational drug (lanadelumab or other drug) or device trial at index date.
Participant with no documented HAE attacks in the 12 months prior to index date.
Trial population
Paediatric participants who have been diagnosed with HAE-C1INH in Germany, France, United Kingdom (UK), Israel, Argentina and Serbia.
Trial design
Design model
Cohort
Time perspective
Retrospective
Treatments tested in this trial
No intervention
Other interventionThis is a non-interventional study.
Treatment groups
40 Participants
are divided into 1 treatment groupGroup A: Participants with HAE-C1INH1 intervention
Trial outcomes
Primary outcomes
1
Percentage of Participants who are Free of HAE Attacks
Percentage of participants who are free of HAE attacks will be reported. A HAE attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx).
Time frame
Up to 13 monthsSecondary outcomes
1
HAE Attack Rate of Participants
Time frame
Up to 13 months2
HAE Attack-free Rate of Participants Relative to Prior Treatment
Time frame
Up to 13 months3
Association Between Covariates of Interest and Attack Free Rate
Association between covariates of interest (e.g., age, sex, family history) and attack-free rate will be determined. Associations between attack free rate and covariates of interest will be modelled, using generalized linear models.
Time frame
Up to 13 months4
Number of Participants With HAE Attack Based on Attack Severity
Number of participants with HAE attack based on attack severity (mild, moderate, severe, unknown) will be reported.
Time frame
Up to 13 months Source ClinicalTrials.gov