RecruitingPHASE2INTERVENTIONAL

A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia

This research study is looking at a new medicine called luspatercept for adults and young people with a specific type of inherited blood disorder called alpha-thalassaemia, specifically the type known as HbH disease. Alpha-thalassaemia can cause anaemia, meaning your blood doesn't carry enough oxygen. The main goal for adults is to see if luspatercept, along with their usual care, helps to improve their anaemia compared to a dummy medicine (placebo). For young people taking part, the study will focus on understanding how safe the medicine is and how it acts in their bodies. The overall aim is to find out if this treatment could be a safe and effective way to help people with alpha-thalassaemia.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Bristol-Myers Squibb

Enrolment target

189

Start

09 Dec 2022

Estimated completion

14 Aug 2034

Anemia

What is this study about?

This study is exploring a new medicine called luspatercept for people living with a blood condition known as alpha-thalassaemia. Specifically, it's for those with 'HbH disease'. Alpha-thalassaemia is an inherited condition that affects how your body makes haemoglobin, which is the part of red blood cells that carries oxygen. This can lead to anaemia, making you feel tired and sometimes needing blood transfusions.

The main purpose of this study is to see if luspatercept can help improve anaemia and reduce the need for blood transfusions in adults with alpha-thalassaemia HbH disease. They will compare it to a dummy treatment (placebo) to understand its effects better. For young people aged 12 to under 18, the study will mainly focus on checking how safe the medicine is for them and how it's handled by their body.

Finding new ways to manage alpha-thalassaemia is important for patients. If this medicine proves to be safe and effective, it could offer a new treatment option, potentially reducing the need for frequent blood transfusions and improving the quality of life for those affected by this condition.

Key takeaways

This study is testing a new medicine, luspatercept, for alpha-thalassaemia HbH disease.
It aims to see if the medicine can reduce anaemia and the need for blood transfusions.
Both adults (18+) and young people (12 to under 18) can take part.
Some participants will receive the new medicine, while others will receive a dummy medicine (placebo).
Participation involves strict eligibility rules, including specific blood test results and health checks.

Who may be eligible?

To join this study, adults must be 18 or older and have a confirmed diagnosis of alpha-thalassaemia HbH disease. They might be needing regular blood transfusions, or perhaps they don't, but still have low blood levels (anaemia).

Young people aged 12 to under 18 can also take part if they have a confirmed diagnosis of alpha-thalassaemia HbH disease. Like adults, they might be either regularly receiving blood transfusions or have anaemia without needing frequent transfusions.

There are also some specific medical conditions that would mean someone couldn't join the study, such as other types of blood disorders or anaemia caused by things like diet. The study also checks your general health to make sure it's safe for you to participate.

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 12 years old or older?
Do you have a diagnosis of alpha-thalassaemia HbH disease?
If you're an adult, have you received at least 6 blood transfusions in the last 24 weeks OR do you have low blood levels (Hb ≤ 10 g/dL) and haven't had a transfusion for at least 8 weeks?
If you're a young person, have you had at least 4 blood transfusions in the last 24 weeks OR do you have low blood levels (Hb ≤ 10 g/dL) and haven't had a transfusion for at least 8 weeks?
Are you generally in good health otherwise?

Answer every question to see your result.

What does participation involve?

The information provided only briefly mentions the purpose and who can join, but doesn't detail what taking part involves. Typically, in studies like this, participants would have regular hospital visits for blood tests, physical examinations, and to receive the study medicine (either luspatercept or a placebo). There would also be check-ups to monitor your health and any side effects. The total duration of participation and how many visits are needed would be explained in full detail by the study team.

Potential risks and benefits

Taking part in a clinical trial might offer benefits like access to a new treatment before it's widely available and closer monitoring of your health. However, there are also potential risks, as the study medicine might have side effects that aren't fully known yet. You also might receive a placebo, meaning you wouldn't get the active medicine. It's important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (36)

Local Institution - 0008
Verified postcode
Halifax, Canada· Withdrawn
Sun Yat-sen Memorial Hospital, Sun Yat-Sen University
Verified postcode
Guangzhou, China· Recruiting
Nanfang Hospital of Southern Medical University
Verified postcode
Guangzhou, China· Recruiting
The First People's Hospital of Foshan
Verified postcode
Foshan, China· Recruiting
Maoming People's Hospital
Approximate
Maoming Shi, China· Recruiting
Shenzhen Second People's Hospital
City only
Shenzhen Shi, China· Recruiting
Liuzhou People's Hospital
Verified postcode
Liuzhou, China· Recruiting
People's Liberation Army The 923rd Hospital
Verified postcode
Nanning, China· Recruiting
Local Institution - 0011
Verified postcode
Haikou, China· Active not recruiting
Local Institution - 0012
Verified postcode
Kunming, China· Completed
Hainan General Hospital
Verified postcode
Haikou, China· Recruiting
The First Affiliated Hospital of Guangxi Medical University
Verified postcode
Nanning, China· Recruiting

Common questions

What is alpha-thalassaemia HbH disease?

It is an inherited blood condition that affects how your body makes red blood cells, leading to anaemia.

What is luspatercept?

It's a new medicine being tested to see if it can help improve anaemia in people with alpha-thalassaemia.

What is a placebo?

A placebo is adummy medicine that looks like the real treatment but doesn't contain any active drug. It helps researchers compare the effects of the new medicine.

What does 'transfusion-dependent' mean?

It means someone regularly needs blood transfusions to manage their condition.

Can children take part in this study?

Yes, young people aged 12 to under 18 years can also participate in this study.

How to find out more

BMS Clinical Trials Contact Center www.BMSClinicalTrials.com

clinical.trials@bms.com 855-907-3286 Official trial record

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