Study on the Safety and Effects of Efpegerglucagon in Children Aged 2 and Older with Congenital Hyperinsulinism

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What is this study about?

This clinical trial is focused on studying a condition known as Congenital Hyperinsulinism (CHI). This is a rare condition where the body produces too much insulin, leading to low blood sugar levels. The trial will test a new treatment called HM15136, which is a solution for injection. The purpose of the study is to evaluate the safety and how well the body tolerates this new treatment, as well as to understand how the drug behaves in the body over time.

Participants in the study will receive the HM15136 treatment for a period of 8 weeks. During this time, researchers will monitor the participants to see how their bodies respond to the treatment. This includes checking for any side effects and measuring how the drug is processed in the body. The study will also look at how the treatment affects the frequency of low blood sugar events in participants.

The study is open-label, meaning both the researchers and participants will know that HM15136 is being administered. Participants will receive the treatment through injections under the skin. The study aims to provide valuable information on the potential benefits and risks of HM15136 for children with Congenital Hyperinsulinism, which could lead to better management of this condition in the future.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing medical history and current treatments for congenital hyperinsulinism (CHI).

Blood tests and other evaluations are performed to ensure the participant meets the study criteria, such as stable therapy with current medications and specific blood glucose levels.

2 treatment initiation

The participant begins treatment with HM15136, a solution for injection administered subcutaneously using a pre-filled syringe.

The treatment is administered over an 8-week period, with injections synchronized with any existing long-acting somatostatin analog treatments if applicable.

3 monitoring and evaluation

Throughout the 8-week treatment period, regular monitoring is conducted to evaluate safety and tolerability. This includes tracking any adverse events, changes in vital signs, and laboratory test results.

Pharmacokinetic assessments are performed to understand how the body processes the medication, including measuring the concentration of the drug in the blood at various times.

4 end of core treatment period

At the end of the 8-week period, a comprehensive evaluation is conducted to assess the overall impact of the treatment on the participant’s condition.

The participant’s response to the treatment is reviewed, including any changes in the frequency of hypoglycemia events.

5 optional extension treatment

If no significant adverse effects are observed, the participant may be offered the opportunity to continue with an optional extension treatment period.

This decision is based on the investigator’s assessment and the participant’s overall health status.

Who Can Join the Study?

Participants must have Congenital Hyperinsulinism (CHI), which is a condition where the body makes too much insulin, leading to low blood sugar.
Participants must have ongoing low blood sugar, experiencing at least 3 episodes per week, even with current standard treatments.
Participants should be on stable treatment with standard medications or not responding to them, with or without extra nutritional support like special feeding methods.
Participants aged 12 and older should be on stable treatment with certain medications like diazoxide or somatostatin analogs for at least 3 months before joining the study.
Children aged 2 to 11 should be on stable treatment with diazoxide or octreotide for at least 1 month before joining the study.
Participants using long-acting somatostatin analogs must have been on stable monthly injections for at least 3 months before joining the study.
Participants must have an HbA1c level, which is a measure of blood sugar control, of less than 7%.
Female participants who can have children must not be pregnant or breastfeeding and must use effective birth control during the study and for 60 days after the last dose. Male participants must agree to use condoms during the study and for 60 days after the last dose.
Participants must agree to the study after receiving information about it. For children, one or both parents or guardians must give permission. Adults must provide their own consent unless they cannot, in which case they will not be included in the study.
If participants do not have any significant side effects during the initial 8-week treatment, they may have the option to continue with an extended treatment period.

Who Cannot Join the Study?

Patients who have any other serious health conditions that could interfere with the study.
Patients who are currently participating in another clinical trial.
Patients who have had a recent surgery or are planning to have surgery during the study period.
Patients who are pregnant or breastfeeding.
Patients who have a history of allergic reactions to similar medications.
Patients who are unable to follow the study procedures or attend the required visits.
Patients who have a history of drug or alcohol abuse.
Patients who have certain heart conditions that could be affected by the study medication.
Patients who have uncontrolled high blood pressure.
Patients who have a history of certain mental health disorders.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Otto Von Guericke Universitaet Magdeburg	Magdeburg	Germany

Trial status

Country	Status	Recruitment Start
Germany	Recruiting	30.12.2021

Trial locations

Investigated drugs:

Efpegerglucagon

HM15136 is a medication being studied for its potential to help people with congenital hyperinsulinism, a condition where the body produces too much insulin. This study is looking at how safe and tolerable HM15136 is for patients, as well as how the body processes the medication. The trial involves giving HM15136 to participants over a period of 8 weeks to see how it affects their condition.

Congenital Hyperinsulinism – Congenital Hyperinsulinism is a condition characterized by excessive insulin production by the pancreas, leading to low blood sugar levels. This condition is present from birth and can cause symptoms such as irritability, poor feeding, and lethargy in infants. As the disease progresses, it may lead to more severe symptoms like seizures or developmental delays if not managed properly. The underlying cause is often genetic mutations affecting insulin regulation. The severity and frequency of hypoglycemic episodes can vary among individuals. Management typically involves monitoring blood sugar levels and dietary adjustments to prevent hypoglycemia.

Trial ID:

2024-515290-98-00

Protocol code:

HM-GCG-201

NCT ID:

NCT04732416

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Safety and Effects of Efpegerglucagon in Children Aged 2 and Older with Congenital Hyperinsulinism

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?