A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries
This study is about a rare condition called Hereditary Angioedema (HAE). HAE causes sudden, temporary swelling in different parts of the body. This particular study focuses on children aged 2-11 who have a specific type of HAE (called HAE-C1INH deficiency) and are already taking a medicine called lanadelumab (also known as Takhzyro) to help prevent these swelling attacks. Researchers want to understand how well lanadelumab is working for these children in their everyday lives. They will look at existing medical records to see how long children go without HAE attacks while on the medicine. They will also learn about its safety and how often children need to see a doctor because of their HAE. No new treatments or interventions will be given as part of this study; all information comes from past medical notes.
At a glance
What is this study about?
This study is focused on children who have a rare condition called Hereditary Angioedema (HAE), specifically a type where the body doesn't make enough of a protein called C1 inhibitor. HAE causes sudden and often painful swelling under the skin or inside the body, such as in the tummy, throat, or private areas. This swelling is different from allergies because it doesn't cause itching or hives.
Many children with this type of HAE take a medicine called lanadelumab (you might know it as Takhzyro) to help prevent these swelling attacks from happening. This study wants to understand how effective lanadelumab really is in the day-to-day lives of these children. Researchers will look at existing medical records to see how long children taking lanadelumab go between HAE attacks. They also want to understand the general health of these children, how safe the medicine appears, and how often they need to visit doctors or hospitals because of their HAE.
It's important to know that no new treatments will be given in this study. Researchers will simply look at information that is already in children's medical files. This means there are no extra appointments, tests, or medicines involved beyond the care your child is already receiving. The findings from this study will help doctors and families better understand how lanadelumab helps children with HAE in the real world.
Key takeaways
- This study helps us understand how a medicine already in use, Takhzyro (lanadelumab), works for children with a rare swelling condition (HAE).
- It focuses on children aged 2-11 who are already taking Takhzyro to prevent swelling attacks.
- Researchers will review existing medical records to learn about attack frequency, safety, and healthcare use.
- No new treatments, tests, or appointments are involved, only past medical data is used.
- The findings will help improve care and understanding for children with HAE in the future.
Who may be eligible?
For a child to be included in this study, they would need to meet a few specific requirements. Firstly, they must be between 2 and 11 years old when they first started taking the medicine lanadelumab, and expected to have at least six months of medical information recorded after starting it before they turn 12. Secondly, a doctor must have officially diagnosed them with the specific type of HAE called HAE-C1INH deficiency. Thirdly, they must have started taking lanadelumab as a long-term preventative treatment during a specific time period set by the study.
Also, your child's medical records must clearly show they had HAE attacks both before and after they started taking lanadelumab. This helps researchers compare how often attacks happened before and after starting the medicine. Before joining, either you or your child (if they are old enough and local rules require it) would need to agree to their medical records being used for the study.
Some children would not be included. For example, if your child was already taking part in another study that involved a new medicine (either lanadelumab or another drug) or a new medical device at the time they started this study. Also, if your child hadn't had any HAE attacks in the 12 months before they started taking lanadelumab, they would not be eligible for this study.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is your child aged between 2 and 11 years?
- Has a doctor officially diagnosed your child with HAE due to low C1 inhibitor protein levels?
- Is your child currently taking a medicine called Takhzyro (lanadelumab) to prevent HAE attacks?
- Does your child's medical record show they had HAE attacks both before and after starting Takhzyro?
- Has your child not been involved in another new drug or device study recently?
What does participation involve?
If your child's existing medical records are chosen for this study, there is no active participation required from you or your child. This isn't a study where your child will be given a new medicine or asked to visit a clinic for extra tests. Instead, researchers will be looking back at medical information, like doctor's notes, hospital visits, and details about their HAE attacks, that already exists in their health records. They will be gathering this information to understand how lanadelumab has been working for children with HAE over time. This means that for participants, there are no extra appointments, no changes to their current medication, and no additional follow-up required beyond their usual medical care.
Potential risks and benefits
Locations (24)
- Hospital de Ninos Sor Maria LudovicaVerified postcodeLa Plata, Argentina· Not yet recruiting
- Centro de Alergia e Inmunologia Clinica Mar del PlataVerified postcodeMar del Plata, Argentina· Not yet recruiting
- Hospital de Pediatria S.A.M.I.C.- Prof. Dr. Juan P. GarrahanUnverifiedCiudad de Buenos Aires, Argentina· Not yet recruiting
- Hospital Italiano de Buenos AiresVerified postcodeBuenos Aires, Argentina· Recruiting
- Sanatorio de la Canada-CordobaVerified postcodeCórdoba, Argentina· Recruiting
- AP-HM- Hopital de La TimoneVerified postcodeMarseille, France· Recruiting
- CHU de Grenoble Alpes - Hopital Couple-EnfantVerified postcodeLa Tronche, France· Recruiting
- AP-HP - Hopital Armand TrousseauVerified postcodeParis, France· Recruiting
- Universitatsklinikum UlmVerified postcodeUlm, Germany· Not yet recruiting
- Universitatsklinikum Frankfurt am MainVerified postcodeFrankfurt am Main, Germany· Not yet recruiting
- Hamophilie Zentrum Rhein Main GmbHVerified postcodeFrankfurt am Main, Germany· Recruiting
- Universitatsklinikum MunsterVerified postcodeMünster, Germany· Recruiting
Common questions
What is Hereditary Angioedema (HAE)?
HAE is a rare genetic condition that causes sudden and unpredictable swelling attacks in different parts of the body, like the face, hands, feet, or internal organs, but without itching or hives.
What is lanadelumab (Takhzyro)?
Lanadelumab, also known as Takhzyro, is a medicine used to help prevent swelling attacks in people with Hereditary Angioedema (HAE).
Will my child receive any new medication in this study?
No, this study only looks at existing medical records. Your child will not receive any new medication or intervention as part of this study.
What kind of information will researchers look at?
Researchers will look at information already in your child's medical records, such as their HAE diagnosis, when they started lanadelumab, details about their HAE attacks, and any hospital visits or doctor appointments related to their condition.
How long will my child be involved in the study?
Your child is not actively involved in the study. Researchers will simply collect information from their existing medical records. There's no extra time commitment for you or your child.
How to find out more
Takeda Contact
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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