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HM15136 (Efpegerglucagon) Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinism (CHI)

This study is investigating a new medication, HM15136, for individuals aged 2 years and older who have congenital hyperinsulinism (CHI). CHI is a rare condition where the body produces too much insulin, leading to dangerously low blood sugar levels. Even with their current treatments, some people with CHI still experience low blood sugar episodes. This study will give HM15136 once a week alongside their usual care to see if it's safe, how well the body handles it, and if it helps control blood sugar better. The researchers hope to understand the best way to use this potential new treatment for different age groups.

Em resumo

Status
Ativo, mas não recrutando
Fase
FASE2
Patrocinador
Hanmi Pharmaceutical Company Limited
Meta de inscrição
17
Iniciar
05 May 2022
Conclusão estimada
01 de maio de 2027

Sobre o que é este estudo?

Congenital hyperinsulinism (CHI) is a rare condition where the body makes too much insulin. Insulin is a hormone that helps your body use sugar for energy. Making too much insulin causes blood sugar levels to drop very low, which can be dangerous and lead to serious health problems if not well-managed. Even with standard treatments, some people, including young children, continue to have low blood sugar episodes, which can be quite worrying for them and their families.

This study is looking at a new medicine called HM15136. It's hoped that HM15136 could help manage blood sugar levels more effectively in people with CHI when added to their current treatment. The study aims to understand how safe the medicine is, how well people tolerate it, and if it makes a real difference in controlling blood sugar. This is a "Phase 2" study, which means it's an early stage of testing new medications, focusing on safety and finding the right dose.

The researchers will give HM15136 once a week to participants aged 2 years and older for eight weeks. They will carefully monitor participants to see how the medicine works in their bodies and if it helps to keep blood sugar levels more stable. The goal is to gather information that will hopefully lead to more effective treatment options for people living with CHI.

Pontos principais

  • This study is for people aged 2 and older with congenital hyperinsulinism (CHI).
  • It tests a new medicine, HM15136, to help control low blood sugar.
  • HM15136 is given once a week for 8 weeks alongside current treatments.
  • The main goals are to check safety, how well it's tolerated, and if it helps manage blood sugar.
  • Participants will have regular health checks and blood tests.

Quem pode ser elegível?

To be considered for this study, you or your child must be at least 2 years old and have a diagnosis of congenital hyperinsulinism (CHI). It's important that you are still experiencing episodes of low blood sugar despite already receiving your usual care for CHI.

Your current CHI treatment, which might include medication or nutritional support, needs to be stable. This means no recent changes in your regular medicines or diet for CHI. People who have had a near-total pancreatectomy (a surgery to remove part of the pancreas) or those who are managing their CHI without surgery because it wasn't an option for them can be included. Your long-term blood sugar control, measured by a test called HbA1c, should be below 7%.

However, some conditions would mean you cannot join the study. For example, if you have type 1 or type 2 diabetes, or if your low blood sugar is caused by other medical reasons or certain medications. You also can't have received continuous intravenous glucose or glucagon infusions within the last three months, or be taking certain drugs that might interfere with the study medication or affect your blood sugar levels.

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.

  1. Are you or your child at least 2 years old?
  2. Do you have congenital hyperinsulinism (CHI)?
  3. Are you still experiencing low blood sugar despite your current CHI treatment?
  4. Is your existing CHI treatment stable (no recent changes)?
  5. Do you have other conditions like type 1 or 2 diabetes or other causes for low blood sugar?
Responda a todas as perguntas para ver o seu resultado.

O que envolve a participação?

If you decide to take part, you or your child will receive the study medicine, HM15136, once a week for eight weeks. This medicine will be given in addition to your current regular treatment for congenital hyperinsulinism. During the study, you'll have regular visits with the study team. These visits will involve health checks, blood tests to monitor your blood sugar and how your body is responding to the medicine, and discussions about how you're feeling and any side effects you might experience. The total duration of active treatment and follow-up will be carefully explained to you by the study team, along with all the assessments involved.

Riscos e benefícios potenciais

Participating in this study might offer potential benefits, such as gaining access to a new experimental treatment that could help manage your congenital hyperinsulinism more effectively. However, as with any new medication, there are potential risks, including side effects from HM15136 which are not yet fully known. The study team will explain all known and potential risks in detail, and your health will be closely monitored throughout. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locais (7)

  • The Children's Hospital of Philadelphia
    Código postal verificado
    Filadélfia, Estados Unidos
  • Otto-von-Guericke-Universitaet Magdeburg
    Código postal verificado
    Magdeburg, Germany
  • Hadassah Medical Center (HMC)
    Código postal verificado
    Jerusalem, Israel
  • Pusan National University Yangsan Hospital
    Código postal verificado
    Pusan, South Korea
  • Asan Medical Center
    Código postal verificado
    Seoul, South Korea
  • Great Ormond Street Hospital (GOSH) for Children NHS Foundation Trust
    Código postal verificado
    Londres, Reino Unido
  • Central Manchester University Hospitals NHS Foundation Trust - Royal Manchester Children's Hospital - Centre for Paediatrics and Child Health
    Código postal verificado
    Manchester, Reino Unido

Perguntas frequentes

What is congenital hyperinsulinism (CHI)?

CHI is a rare condition where the body produces too much insulin, leading to dangerously low blood sugar levels.

What is HM15136?

HM15136 is an investigational new medicine being tested to help manage low blood sugar in people with CHI.

How often would I take the study drug?

The study drug, HM15136, would be given once a week for a period of eight weeks.

Will I stop my current CHI treatment if I join?

No, HM15136 will be given as an 'add-on' therapy, meaning you will continue your usual CHI treatment alongside it.

What does 'Phase 2' mean for a clinical trial?

Phase 2 means the study is in an early stage, focusing on assessing the safety and effectiveness of the new medicine and finding the right dose.

Como saber mais

Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.

Discussão

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