Active not recruitingPHASE2INTERVENTIONAL

Apotransferrin in Atransferrinemia

This study is for people who have a very rare health problem called atransferrinemia. This condition means your body doesn't make enough of a protein called transferrin, which is important for your body. Right now, there isn't a regular treatment for it. This study wants to find out if a new medicine, called human apotransferrin, can help. Researchers will check how the medicine moves through the body, how effective it is in treating the condition, and if it has any side effects. The aim is to find a safe and helpful new treatment for people living with atransferrinemia, potentially improving their health and quality of life.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Prothya Biosolutions

Enrolment target

Start

01 Dec 2010

Estimated completion

01 Jan 2028

Congenital Atransferrinemia

What is this study about?

This study is looking into a very rare inherited condition called atransferrinemia. People with this condition have a problem where their body doesn't produce enough of a vital protein called transferrin. This protein is essential for carrying iron around the body, and without enough of it, serious health problems can occur. Currently, there isn't a standard, regular treatment for people with atransferrinemia, which means managing the condition can be very challenging.

The main goal of this research is to see if a new medicine, called apotransferrin, can be a helpful and safe treatment option. Researchers will carefully investigate how this medicine works inside the body – for example, how long it stays in the system and how it affects the body's iron levels. They will also be looking closely at whether the treatment improves the health of people with atransferrinemia and if there are any side effects.

The findings from this study are very important. If apotransferrin is found to be effective and safe, it could become the first regular treatment available for atransferrinemia. This would be a significant step forward, offering new hope and potentially a much better quality of life for individuals and families affected by this rare condition.

Key takeaways

This study is for a very rare iron-related condition called atransferrinemia.
It's testing a new medicine, human apotransferrin, as a potential treatment.
The aim is to check if the medicine is safe and if it helps people with the condition.
There is currently no regular treatment available for atransferrinemia.
Participating involves regular health checks and taking the study medicine.
You can stop taking part in the study at any time.

Who may be eligible?

To join this study, you must have a confirmed diagnosis of atransferrinemia. This means that a doctor has officially told you that you have this condition, and blood tests show your transferrin levels are below a certain mark (under 40 mg/dl). You also need to be able to understand and sign a consent form, which explains all about the study in detail.

There are some reasons why you might not be able to join. For example, if you know you have allergic reactions to human plasma or products made from plasma (like some blood products), or if you have certain antibodies in your blood that could cause problems with the treatment, you wouldn't be able to take part.

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.

Have I been told by a doctor that I have atransferrinemia?
Do my blood tests show my transferrin levels are low (under 40 mg/dl)?
Am I able to understand information about the study and agree to take part?
Do I have any allergies to human plasma or plasma products?
Have I been told I have anti-immunoglobulin A antibodies in my blood?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, you will be given the study medicine, human apotransferrin. The study team will then closely monitor you to see how your body reacts to the treatment. This will involve regular visits to the clinic where you will have check-ups, blood tests, and other assessments to measure how the medicine is working and to check for any side effects. The team will explain exactly how often these visits will be and what each visit will involve.

After you stop taking the main study medicine, there might be some follow-up appointments to make sure you are doing well and to complete all the necessary checks. The total time you would be involved in the study, from your first visit to your last follow-up, will be clearly explained to you by the study team.

Potential risks and benefits

Taking part in this study could potentially offer significant benefits, as it aims to find the first regular treatment for atransferrinemia, which could improve your health. However, like all medicines, apotransferrin might cause side effects, and some tests might be uncomfortable or carry small risks. The study team will explain all known potential side effects and risks to you in detail. Remember, joining this study is completely your choice, and you are free to withdraw at any time without giving a reason, and it will not affect your usual medical care.

Locations (3)

Klinikum Aschaffenburg
Verified postcode
Aschaffenburg, Germany
AO San Gerardo
Verified postcode
Monza, Italy
Vall d'Hebron Hospital
Verified postcode
Barcelona, Spain

Common questions

What is atransferrinemia?

It's a very rare inherited condition where your body doesn't make enough of a protein called transferrin, which carries iron.

What is apotransferrin?

This is the medicine being tested in the study; it's a replacement for the missing transferrin protein.

Will I get 'real' medicine or a dummy treatment?

This study is testing a specific medicine, apotransferrin, so participants will receive this active treatment.

How long will the study last for me?

The total duration of your participation, including treatment and follow-up, will be explained by the study team.

Can I stop participating if I change my mind?

Yes, you can leave the study at any time, for any reason, without it affecting your medical care.