Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

Evaluation of the efficacy and safety of empagliflozin in the treatment of neutropenia in patients with glycogenosis Ib. EMPAtia.

This research is investigating a medicine called empagliflozin, also known as Jardiance, for adults living with a very rare inherited condition called Glycogenosis type Ib. People with this condition often have a problem called neutropenia, which means they have too few white blood cells called neutrophils, making them more prone to infections. The main goal of this study is to carefully check if empagliflozin is safe to use and how well people tolerate it. Researchers will also look to see if the medicine helps to bring the number and function of these important neutrophil cells back to normal. This is an early-stage study, meaning it's one of the first times this medicine is being tested for this specific condition.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Instytut Pomnik Centrum Zdrowia Dziecka

Enrolment target

Start

02 Dec 2024

Glycogenosis type Ib is an ultra-rare disease inherited in an autosomal recessive manner.

What is this study about?

Imagine your body's immune system as a strong army protecting you from illness. Part of that army are special soldiers called neutrophils, which are a type of white blood cell that fights off infections. For people with a very rare illness called Glycogenosis type Ib (often shortened to GSD Ib), their body doesn't make enough of these neutrophil soldiers, or the ones they do have don't work properly. This makes them much more likely to get serious infections. GSD Ib is a condition you are born with and it runs in families.

This study is looking into a medicine called empagliflozin, which you might know by its brand name Jardiance. This medicine is currently used for other conditions, but doctors believe it might also help people with GSD Ib by improving their neutrophil levels. Because GSD Ib is so rare, finding new treatments is very important.

The main aim of this study is to carefully check how safe empagliflozin is for people with GSD Ib and if there are any side effects. Researchers will also be looking to see if the medicine can help increase the number of those important white blood cells (neutrophils) and make them work better. This is an early study, so it's about exploring if this medicine could be a promising new option for people with GSD Ib in the future.

Key takeaways

This study is testing a medicine called empagliflozin (Jardiance) for rare Glycogenosis type Ib.
It aims to see if the medicine is safe and can increase infection-fighting white blood cells (neutrophils).
It's an early-stage study, meaning researchers are exploring a potential new treatment.
Participation involves taking tablets and having regular health check-ups.
This research could help improve treatment options for GSD Ib in the future.

Who may be eligible?

To be considered for this study, you must be an adult, 18 years old or older. There is no upper age limit for taking part.

This study is open to both men and women.

Because this is a specific study for Glycogenosis type Ib, you would need to have a confirmed diagnosis of this rare condition to be part of the research.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a confirmed diagnosis of Glycogenosis type Ib?
Are you comfortable taking daily medication as prescribed?
Are you able to attend regular clinic visits for monitoring?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be given the study medicine, Jardiance (empagliflozin), as tablets. You would have regular visits to the clinic where the research team would monitor your health very closely. During these visits, doctors and nurses would perform various checks, which might include blood tests to look at your neutrophil levels and other general health markers. They will also ask you about how you are feeling and if you've experienced any side effects.

The research team will collect all this information to understand how safe the medicine is and if it's helping. You would also have follow-up appointments after you stop taking the medicine to ensure your health is stable. The total length of your participation in the study, including follow-up, would be discussed with you by the study team.

Potential risks and benefits

Participating in this study might offer a potential benefit of receiving a new medicine that could help improve your condition, specifically by boosting your neutrophil levels and reducing infection risk. However, like all medicines, empagliflozin can have side effects, and the researchers will be carefully monitoring for these. The study aims to understand what these potential risks are for people with GSD Ib. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Poland

Common questions

What is Glycogenosis type Ib?

It's a very rare inherited illness where your body has trouble processing sugars, which can lead to low numbers of infection-fighting white blood cells (neutrophils).

What is empagliflozin (Jardiance)?

It's a medicine currently used for other health conditions. This study is exploring if it can help improve neutrophil levels in people with Glycogenosis type Ib.

Why is this study important?

Because Glycogenosis type Ib is very rare, finding new and effective treatments is crucial to improve the health and quality of life for people living with it.

Will I have to pay to be part of this study?

No, you will not have to pay for the study medicine or any study-related visits or tests.

What if I experience side effects during the study?

The study team will monitor you closely for any side effects, and you should tell them immediately if you feel unwell. They are there to support you.