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Efpegerglucagon

Efpegerglucagon, also known as HM15136, is being studied in clinical trials as a potential treatment for children aged 2 and older with Congenital Hyperinsulinism (CHI). This rare genetic condition causes excessive insulin production, leading to dangerously low blood sugar levels. The ongoing research aims to evaluate the safety, tolerability, and effectiveness of Efpegerglucagon in managing CHI symptoms and improving patients’ quality of life.

Table of Contents

What is EFPEGERGLUCAGON?

EFPEGERGLUCAGON, also known by its product code HM15136, is a new medication being studied for the treatment of Congenital Hyperinsulinism (CHI) in children aged 2 years and older[1]. It is a solution for injection that comes in a pre-filled syringe and is administered subcutaneously (under the skin)[1].

Understanding Congenital Hyperinsulinism (CHI)

Congenital Hyperinsulinism (CHI) is a rare genetic disorder that affects the body’s regulation of blood sugar levels[1]. In CHI, the pancreas produces too much insulin, leading to frequent episodes of low blood sugar (hypoglycemia). This condition can be dangerous, especially for young children, as it can cause seizures, brain damage, and other serious complications if not properly managed.

How EFPEGERGLUCAGON Works

While the exact mechanism of action is not fully described in the provided information, EFPEGERGLUCAGON is likely designed to help regulate blood sugar levels in patients with CHI. It may work by counteracting the effects of excess insulin or by helping to maintain more stable glucose levels in the blood[1].

Clinical Trial Details

A Phase 2 clinical trial, called ACHIEVE, is being conducted to evaluate EFPEGERGLUCAGON[1]. This trial aims to:

  • Assess the safety and tolerability of EFPEGERGLUCAGON
  • Study how the drug is processed in the body (pharmacokinetics)
  • Evaluate its effectiveness in reducing hypoglycemic events

The trial consists of an 8-week core treatment period, followed by an optional 44-week extension period for long-term evaluation[1].

Eligibility Criteria

To participate in the EFPEGERGLUCAGON trial, patients must meet certain criteria, including:

  • Be at least 2 years old
  • Have a confirmed diagnosis of CHI
  • Experience at least 3 hypoglycemic events per week (blood glucose <70 mg/dL or <3.9 mmol/L)
  • Be on stable therapy with current standard-of-care medications or have documented non-response to these treatments

There are also several exclusion criteria, such as having type 1 or type 2 diabetes, severe anemia, or certain other medical conditions[1].

Safety and Side Effects

As this is an ongoing clinical trial, the full safety profile of EFPEGERGLUCAGON is not yet known. The study is closely monitoring for any adverse events (side effects) that may occur during treatment. Some areas being evaluated include:

  • Changes in laboratory test results
  • Effects on vital signs (blood pressure, heart rate, etc.)
  • Changes in ECG parameters, particularly the QT interval
  • Immune system responses to the medication

Patients and caregivers should report any unusual symptoms or side effects to their healthcare providers immediately[1].

Potential Benefits

If successful, EFPEGERGLUCAGON could offer several potential benefits for patients with CHI:

  • Reduction in the frequency and severity of hypoglycemic events
  • Improved quality of life for patients and their families
  • Potentially fewer complications associated with chronic low blood sugar
  • A new treatment option for patients who don’t respond well to current therapies

It’s important to note that as EFPEGERGLUCAGON is still in clinical trials, its effectiveness and long-term benefits are yet to be fully determined[1].

Aspect Details
Drug Name Efpegerglucagon (HM15136)
Condition Studied Congenital Hyperinsulinism (CHI)
Trial Phase Phase 2
Age Group 2 years and older
Administration Subcutaneous injection
Primary Objectives Safety, tolerability, and pharmacokinetics
Secondary Objectives Reduction in hypoglycemia events, long-term safety
Treatment Duration 8 weeks (core period) + 44 weeks (optional extension)
Key Inclusion Criteria CHI diagnosis, ≥3 hypoglycemia events/week, stable on standard therapy
Key Exclusion Criteria Type 1/2 diabetes, serious adverse reactions to study drug, significant lab abnormalities

FAQ

  • What is Congenital Hyperinsulinism (CHI)? Congenital Hyperinsulinism (CHI) is a rare genetic condition where the body produces too much insulin, leading to frequent episodes of low blood sugar (hypoglycemia). This can be dangerous, especially for children, and may cause developmental issues if not properly managed.
  • Who is eligible for the Efpegerglucagon clinical trial? The trial is open to male and female patients aged 2 years and older with CHI who experience at least 3 episodes of low blood sugar (below 70 mg/dL) per week, despite current standard treatments. Participants must be on stable therapy or documented as non-responsive to standard care medications.
  • How is Efpegerglucagon administered in the trial? Efpegerglucagon is administered as a subcutaneous injection (under the skin) using a pre-filled syringe. The exact dosing schedule and amounts are determined by the study protocol and may vary based on the patient's individual needs.
  • What are the main objectives of the Efpegerglucagon clinical trial? The primary objectives are to evaluate the safety and tolerability of Efpegerglucagon, as well as to study its pharmacokinetics (how the drug moves through the body). Secondary objectives include assessing the drug's effectiveness in reducing the frequency of low blood sugar events and evaluating its long-term safety in an optional 44-week extension period.
  • Are there any potential side effects or risks associated with Efpegerglucagon? As with any clinical trial, there may be potential risks and side effects. The study closely monitors participants for adverse events, including changes in laboratory tests, physical examinations, vital signs, and ECG parameters. Specific side effects are not mentioned in the provided information, as they are being actively studied during the trial.

Ongoing Clinical Trials on Efpegerglucagon

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

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Germany

Glossary

  • Congenital Hyperinsulinism (CHI): A rare genetic disorder characterized by excessive insulin production, leading to frequent episodes of low blood sugar (hypoglycemia).
  • Hypoglycemia: A condition where blood sugar (glucose) levels fall below normal, typically defined as less than 70 mg/dL (3.9 mmol/L).
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Standard of Care (SoC): The currently accepted treatment methods for a particular condition, which may include medications like diazoxide or somatostatin analogs for CHI.
  • Adverse Event (AE): Any unfavorable or unintended sign, symptom, or disease that occurs during a clinical trial, whether or not it is related to the study drug.
  • ECG (Electrocardiogram): A test that measures the electrical activity of the heart, used to monitor for any cardiac effects of the study drug.
  • QTcF: The QT interval on an ECG corrected for heart rate using Fridericia's formula, used to assess potential cardiac effects of medications.
  • Subcutaneous injection: A method of administering medication by injecting it into the layer of tissue between the skin and muscle.
  • Immunogenicity: The ability of a substance to provoke an immune response in the body, which is monitored as a safety measure in clinical trials.
  • Orphan Drug Designation: A special status granted to drugs intended to treat rare diseases, which may provide incentives for their development.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-efpegerglucagon-in-children-aged-2-and-older-with-congenital-hyperinsulinism/