At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- No intervention (other)
- How long the study runs
- Study runs about 5 months (dates as stated)
- About the drug or intervention
- No intervention — other: This is a non-interventional study.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Observing health over time
- Ages
- 2 Years to 11 Years
- Who
- All
- Number of participants
- 37
- Started
- 2026-02-05
- Last checked
- 2026-07
Plain English Summary
What is this study?
- • Testing a new treatment for hereditary angioedema (hae)
- • Clinical study - 37 participants
- • HAE is a rare condition
Who can take part?
- • Ages 2 Years to 11 Years
- • Diagnosed with hereditary angioedema (hae)
Where?
- • Newcastle upon Tyne - Royal Victoria Infirmary
- • London - The Royal London Hospital - PPDS
This is a simplified summary. Always discuss with your doctor before making any decisions.
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 2 Years - 11 Years
- Who can join: All genders
Treatment history
Treatments you must have had:
- ✓ by local regulations)
What the study is looking for
- ✓Participant is aged 2 to less than (\<) 12 years at the time of lanadelumab initiation and is expected to have at...
- ✓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).
- ✓Participant's medical record contains documentation of HAE attacks in the pre-index period and after lanadelumab...
Who cannot take part
- ✗Participant was enrolled in a therapeutic investigational drug (lanadelumab or other drug) or device trial at index...
- ✗Participant with no documented HAE attacks in the 12 months prior to index date.
See the full criteria
Where Is This Study? (2 UK sites)
Royal Victoria Infirmary
Newcastle upon Tyne NE1 4LP, United Kingdom
The Royal London Hospital - PPDS
London E1 1FR, United Kingdom