Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)
This research study is investigating a new medication, Encaleret, for children and teenagers from birth to 17 years old who have a genetic condition called Autosomal Dominant Hypocalcemia Type 1, or ADH1. ADH1 causes low calcium levels in the blood. The main goals of the study are to understand how Encaleret is processed by the body in young people, to check if it effectively improves their symptoms, and to make sure it is safe for them to use. This is a crucial step to potentially offer new treatment options for children living with ADH1. It's a late-stage study, meaning it has already shown some promise in earlier tests.
Em resumo
Sobre o que é este estudo?
This study is focused on a genetic condition called Autosomal Dominant Hypocalcemia Type 1, or ADH1. People with ADH1 have a problem with how their bodies control calcium levels, often leading to low calcium in their blood. This can cause various health issues.
Researchers are testing a new medicine called Encaleret. They want to find out how this medicine works in children and teenagers (from newborns up to 17 years old) who have ADH1. They will be looking at several things: how the body handles the medicine (like how much is absorbed and how long it stays in the system), if it helps to improve the symptoms of ADH1, and if it's safe to use in this age group. This type of research is vital because it could lead to new and better treatments for children with this rare condition.
This study is in its later stages (Phase 2 and Phase 3), which means it has already shown some positive results in earlier tests. The goal now is to gather more detailed information to determine if Encaleret could become a widely available treatment. It's important to understand that participating in a study helps everyone learn more about medical conditions and potential new ways to treat them.
Pontos principais
- This study is testing a new medicine called Encaleret for children aged 0-17 with ADH1.
- The main goals are to check how the medicine works, if it helps symptoms, and if it's safe.
- Participation involves regular clinic visits for medical checks and blood tests.
- There's a chance your child could benefit from a new treatment, but also a risk of side effects.
- Você pode retirar seu filho do estudo a qualquer momento sem afetar o cuidado dele.
Quem pode ser elegível?
To join this study, children and teenagers need to be between birth and 17 years old. They must have a confirmed diagnosis of Autosomal Dominant Hypocalcemia Type 1 (ADH1), or have test results that strongly suggest it, along with symptoms related to low calcium levels.
Participants should also have been receiving their usual ADH1 treatment for at least three to six months before starting the study, depending on the specific group they might be in. If a child is legally able, they will be asked for their agreement to take part. For younger children, a parent or legal guardian will need to give their permission.
There are also some reasons why a child might not be able to join. For example, if they've had certain surgeries like on the thyroid or parathyroid glands, or if they've had specific types of cancer or kidney transplants. Also, if they've recently taken part in other clinical studies or are on certain medications that could interact with the study drug, they might not be eligible. The study team will review each child's medical history carefully to make sure it's safe for them to participate.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- Is my child between newborn and 17 years old?
- Does my child have a confirmed diagnosis of ADH1 or strong evidence of it?
- Does my child experience symptoms related to low calcium levels from ADH1?
- Has my child been on their usual ADH1 treatment for at least 3-6 months?
- Has my child NOT had a thyroid or parathyroid surgery?
O que envolve a participação?
If your child takes part in this study, they will receive the study medication, Encaleret. They will have regular visits to the clinic so the study team can monitor their health, take blood samples to check calcium levels and how their body is handling the medicine, and assess any changes in their ADH1 symptoms. These visits will help doctors understand how well Encaleret is working and if it's safe.
The study involves different groups of participants, and the exact schedule of visits and tests will vary slightly depending on which group your child is in. There will be initial screening tests to ensure your child is suitable for the study, followed by treatment and regular check-ups. The total length of time your child would be involved in the study will be explained by the study team, but it's designed to gather enough information about the long-term effects of the medication.
Riscos e benefícios potenciais
Localizações (6)
- Universidade de YaleCódigo postal verificadoNew Haven, Estados Unidos· Recrutamento
- Children's National Medical CenterCódigo postal verificadoWashington D.C., Estados Unidos· Recrutamento
- Nemours Children's HealthCódigo postal verificadoJacksonville, Estados Unidos· Recrutamento
- The Children's Hospital of PhiladelphiaCódigo postal verificadoFiladélfia, Estados Unidos· Recrutamento
- Royal London HospitalCódigo postal verificadoLondres, Reino Unido· Recrutamento
- Royal Manchester Children's HospitalCódigo postal verificadoManchester, Reino Unido· Recrutamento
Perguntas frequentes
What is Autosomal Dominant Hypocalcemia Type 1 (ADH1)?
ADH1 is a rare genetic condition where the body has trouble controlling calcium levels, usually resulting in low calcium in the blood.
What is Encaleret?
Encaleret is a new investigational medicine being tested to see if it can help improve calcium levels and symptoms in people with ADH1.
Which age group is this study for?
This study is specifically for children and teenagers, from birth (newborns) up to 17 years old.
Will my child definitely get the new medicine?
Yes, if your child is eligible and joins the study, they will receive Encaleret as the study medication.
Can we stop participating at any time?
Yes, you have the right to withdraw your child from the study at any point, and it will not affect their future medical treatment.
Como saber mais
Medical Information
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
Interessado em participar?
Discussão
Discussão da comunidade
Desenvolvido pelo nosso fórum em community.patient.info. Por favor, seja respeitoso — isto não é um conselho médico.