Not yet recruitingPHASE2INTERVENTIONAL

Luspatercept in Patients Affected With Rare Inherited Anemias

This study is testing a medicine called Luspatercept (also known as Reblozyl) in adults who have certain rare inherited blood conditions. These conditions affect how their body makes red blood cells, leading to anaemia, which means they don't have enough healthy red blood cells. The study is particularly looking at three types of these conditions: constitutional non-syndromic sideroblastic anaemia (CSA), constitutional dyserythropoietic anaemias (CDA), and Diamond-Blackfan anaemia (DBA) that doesn't need regular blood transfusions. Researchers want to find out if Luspatercept can help improve blood cell production and reduce the need for blood transfusions in these patients. It's happening in centres in France and Italy and involves about 45 patients.

At a glance

Status

Not yet recruiting

Phase

PHASE2

Sponsor

EuroBloodNet Association

Enrolment target

Start

15 Jan 2026

Estimated completion

30 Sep 2029

Hereditary Red Blood Cell Disorder (Disorder)

What is this study about?

This research study is about a new medicine called Luspatercept, which is also known as Reblozyl. It’s being tested for adults who have rare inherited conditions that affect their red blood cells. These conditions can make it hard for the body to produce enough healthy red blood cells, leading to a problem called anaemia. Anaemia can make you feel tired and weak, and sometimes people with these conditions need regular blood transfusions.

The study aims to see if Luspatercept can help people with these specific conditions produce more healthy red blood cells. The team particularly wants to find out if the medicine can reduce how often people need blood transfusions, or help their bodies make more blood cells on their own. This is an early-stage study (called Phase 2), which means researchers are still learning about how well the medicine works and if it’s safe for these specific rare conditions.

Researchers are focusing on three main types of inherited anaemia: constitutional non-syndromic sideroblastic anaemia (CSA), constitutional dyserythropoietic anaemias (CDA) (types 1 and 2), and Diamond-Blackfan anaemia (DBA) in patients who don't usually need regular blood transfusions. They want to include about 45 patients from hospitals across France and Italy, looking carefully at different genetic types within these conditions.

Key takeaways

This study is for adults with specific rare inherited blood conditions causing anaemia.
It's testing a medicine called Luspatercept (Reblozyl) to see if it helps blood production.
The goal is to improve anaemia and potentially reduce the need for blood transfusions.
It's an early-stage (Phase 2) study taking place in France and Italy.
Only specific types of inherited anaemia are included, and genetic confirmation is needed.

Who may be eligible?

To be part of this study, you need to be an adult, aged between 18 and 99 years old. You must have one of three specific rare inherited blood conditions: constitutional non-syndromic sideroblastic anaemia (CSA), constitutional dyserythropoietic anaemias (CDA) (type I or II), or Diamond-Blackfan anaemia (DBA) where you *don't* need regular blood transfusions.

Your exact diagnosis for these conditions must be confirmed by genetic tests. For CSA and CDA, you might be included whether you currently need regular blood transfusions or not. If you do need transfusions, you'd typically have received 6 to 20 units of blood in the past 24 weeks. If you don't need regular transfusions, you would still have significant anaemia, meaning your blood tests show low haemoglobin levels, and you wouldn't have had many recent transfusions.

Finally, your kidneys and liver need to be working well, as shown by standard blood tests. Your doctor will carefully check all of these points to see if this study could be right for you.

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 one of these diagnosed conditions: constitutional non-syndromic sideroblastic anaemia (CSA), constitutional dyserythropoietic anaemias (CDA), or Diamond-Blackfan anaemia (DBA) where you typically don't need regular blood transfusions?
Has your diagnosis been confirmed by a genetic test?
Are your kidney and liver tests within a healthy range, as determined by your doctor?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be given the study medicine, Reblozyl (Luspatercept). The study is planned to take place in specialised hospitals within France and Italy. You will have regular visits to the hospital for assessment, which will include blood tests to check your blood cell levels, kidney and liver function, and to see if the medicine is helping. The doctors and nurses will closely monitor your health throughout the study. We don’t have exact details here about how long each visit will be, how often you’ll need to visit, or the total duration of the study for each person, but this would be fully explained by the study team if you're eligible and interested.

Potential risks and benefits

Taking part in any medical study has potential benefits and potential risks. A possible benefit of this study is that Luspatercept might help improve your body's ability to make healthy red blood cells, which could reduce your anaemia and potentially lower your need for blood transfusions. However, as this is an early-stage study, we don't yet fully know how well it works or if it works for everyone. There might also be side effects from the medicine, or the treatment might not be effective for you. All possible risks and side effects will be fully explained by the study team. Remember, you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (6)

GCS Groupement des Hôpitaux de l'Institut Catholique de Lille
Verified postcode
Lille, France
Centre Hospitalier Universitaire de Montpellier
Verified postcode
Montpellier, France
Assistance Publique Hôpitaux de Paris - Hôpital Saint-Louis
Verified postcode
Paris, France
Assistance Publique Hôpitaux de Paris - Hôpital Necker
Verified postcode
Paris, France
CHU Bordeaux-Hôpital Haut-Lévêque
Verified postcode
Pessac, France
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano
Verified postcode
Milan, Italy

Common questions

What is anaemia?

Anaemia means your body doesn't have enough healthy red blood cells. This can make you feel very tired, weak, and short of breath.

What are 'inherited anemias'?

These are types of anaemia that you're born with because they're passed down through your family's genes. They affect how your body makes red blood cells.

What is Luspatercept (Reblozyl)?

It's a new medicine being tested. It's designed to help your body produce more red blood cells and might reduce the need for blood transfusions.

What does 'Phase 2 trial' mean?

It means this is an earlier stage of testing for a new medicine. Researchers are looking to see how well it works and if it's safe for people with these specific conditions.

Will I need blood transfusions if I'm in the study?

The study aims to reduce the need for transfusions, but some patients may still need them. Your doctors will monitor your needs closely.

How to find out more

Adeline Gladieux

adeline.gladieux@eurobloodnetassociation.com 0171207059 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.