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Study on the Use of Empagliflozin for Treating Neutropenia in Patients with Glycogen Storage Disease Type Ib

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a very rare inherited disease called Glycogen Storage Disease Type Ib (GSD Ib). This condition affects the body's ability to store and use sugar properly, leading to a shortage of a type of white blood cell called neutrophils, a condition known as neutropenia. Neutrophils are important for fighting infections, so people with GSD Ib can be more prone to infections.

The study is testing a medication called empagliflozin, which is usually used to treat diabetes, to see if it can help increase the number of neutrophils in patients with GSD Ib. The purpose of the study is to evaluate the safety and how well patients tolerate empagliflozin when used for treating neutropenia in GSD Ib. Some participants will receive empagliflozin, while others may receive a placebo. The study will monitor participants over a period to see if empagliflozin helps restore neutrophil numbers and function.

Participants in the study will take the medication in the form of film-coated tablets. The study will involve regular visits to monitor the safety of the treatment and to check for any side effects. The goal is to gather information on how safe empagliflozin is for patients with GSD Ib and whether it can effectively help manage neutropenia in these patients.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, you will be required to provide informed consent. If you are under 13 years of age, your legal representative will provide consent on your behalf. If you are 13 years or older, both you and your legal representative will need to provide consent.

    You must have a confirmed diagnosis of glycogenosis type Ib with neutropenia or abnormal activity in certain blood cells.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to evaluate your current health status and confirm eligibility for the trial. This may include blood tests and other diagnostic procedures.

  3. Step 3

    Medication administration

    You will begin taking the medication Jardiance 10 mg film-coated tablets, which contains the active substance empagliflozin.

    The medication is taken orally, meaning you will swallow the tablet. The dosage and frequency will be determined by the study team and explained to you.

  4. Step 4

    Regular follow-up visits

    You will attend regular follow-up visits throughout the study period. These visits are essential for monitoring your health and the effects of the medication.

    During these visits, safety data will be collected to assess any adverse reactions to the medication.

  5. Step 5

    Evaluation of treatment effects

    The primary goal is to evaluate the safety and tolerability of the treatment. This involves monitoring any side effects you may experience.

    The secondary goal is to assess the effectiveness of the treatment in restoring the number and function of certain blood cells.

  6. Step 6

    Completion of the study

    The study is expected to conclude by March 26, 2025. Upon completion, a final assessment will be conducted to evaluate the overall outcomes of the treatment.

Who can join the trial?

7 criteria

  • Both males and females can participate.
  • Participants must be 4 weeks old or older.
  • Participants must have a diagnosis of Glycogenosis type Ib, which is a rare inherited disease.
  • Participants must have neutropenia (a condition where there are fewer white blood cells called neutrophils, which help fight infections) and/or abnormal NADPH oxidase activity in neutrophils (a specific function of these white blood cells).
  • For children under 13 years old, a legal representative must give consent for them to join the study.
  • For children 13 years and older, both the legal representative and the child must give consent.
  • Adult participants must give their own voluntary informed consent to join the study.

Who cannot join the trial?

8 criteria

  • Patients who are not diagnosed with **Glycogenosis type Ib** cannot participate. This is a specific genetic condition.
  • Patients who are not experiencing **neutropenia** cannot participate. Neutropenia is a condition where there is a low level of neutrophils, a type of white blood cell important for fighting infections.
  • Patients who are not within the specified age range cannot participate. The study includes certain age groups only.
  • Patients who are not willing or able to follow the study procedures cannot participate.
  • Patients who have other medical conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who are participating in another clinical trial cannot participate.
  • Patients who have a history of allergic reactions to the study medication cannot participate.
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Investigated drugs

Empagliflozin is a medication that is being studied for its potential to treat neutropenia in patients with glycogen storage disease type Ib (GSD Ib). Neutropenia is a condition where there is a lower than normal number of neutrophils, a type of white blood cell that helps fight infections. Empagliflozin is usually used to help control blood sugar levels in people with type 2 diabetes, but in this trial, researchers are exploring its safety and how well it works for treating neutropenia in GSD Ib patients. The goal is to see if empagliflozin can help increase the number of neutrophils and improve the patients' ability to fight infections.

What is already known about the treatment

Empagliflozin – Empagliflozin is administered orally in the form of a tablet. It is currently being studied for its potential use in treating neutropenia, a condition characterized by low levels of neutrophils, in patients with glycogen storage disease type Ib, a rare genetic disorder. The medication works by inhibiting a protein in the kidneys called SGLT2, which helps to lower blood sugar levels, but its exact mechanism in treating neutropenia is still under investigation. Empagliflozin is classified as an SGLT2 inhibitor, a type of drug commonly used to manage diabetes.

Investigated diseases

Glycogen storage disease type Ib – Glycogen storage disease type Ib is a genetic disorder that affects the way the body stores and uses glycogen, a form of sugar stored in the liver and muscles. It is inherited in an autosomal recessive manner, meaning both parents must carry a copy of the mutated gene for a child to be affected. This disease is characterized by a deficiency in the enzyme glucose-6-phosphate translocase, which leads to the accumulation of glycogen in the liver and kidneys. Over time, this can cause an enlarged liver, low blood sugar levels, and growth delays. Individuals with this condition often experience neutropenia, a decrease in neutrophils, which are a type of white blood cell important for fighting infections. The progression of the disease can lead to increased susceptibility to infections and other complications related to the immune system.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-512840-52-00Protocol codeEMPAtiaEstimated enrolment20 patientsSponsorInstytut Pomnik Centrum Zdrowia Dziecka

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).