RecruitingPHASE3INTERVENTIONAL

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

This study is checking the long-term safety of a medicine called luspatercept. It's for adults, aged 18 and over, who have blood conditions like Myelodysplastic Syndromes (MDS), Beta-thalassemia, or Myelofibrosis and have already been part of another luspatercept study. If your doctor thinks you're benefiting from luspatercept, or if you were on a placebo but could now benefit from the active medicine, you might be able to join. The main goal is to keep an eye on your health and any side effects over a long period, which could be several years, as you continue with or finish your treatment.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Celgene

Enrolment target

665

Start

12 Aug 2019

Estimated completion

12 May 2028

Myelodysplastic Syndromes (MDS)Beta-thalassemia Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis

What is this study about?

This study is like a continuation for people who have already been taking part in a clinical trial for a medicine called luspatercept. Luspatercept is a treatment for certain blood conditions such as Myelodysplastic Syndromes (MDS), a type of blood cancer; Beta-thalassemia, which is an inherited blood disorder; and Myelofibrosis, where the bone marrow is replaced by scar tissue. The main purpose of this new study is to carefully watch how safe luspatercept is over a much longer period of time.

Taking part in this study means you’ll either continue receiving luspatercept if your doctor thinks it's helping you, or, if you were on a dummy treatment (placebo) in a previous study, you might switch to receiving luspatercept. If you've already finished treatment in a previous study, you might join this one for long-term health checks, to see how you are doing over many years. This helps researchers understand the full picture of the medicine's safety.

By carefully checking on participants for several years, doctors and researchers can learn a lot about any potential long-term side effects or benefits of luspatercept. This information is really important for making sure new medicines are as safe and effective as possible for everyone who might need them in the future. The study will pay close attention to your general health and any changes, including checking for any new health problems that might develop.

Key takeaways

This study is for checking the long-term safety of luspatercept.
It's for people already in a luspatercept study.
You might continue treatment, start it, or just have long-term health checks.
The study involves regular visits and monitoring over several years.
It aims to gather important information about the medicine's effects over time.
Your general health and any new problems will be closely monitored.

Who may be eligible?

To be able to join this study, you must be at least 18 years old and already taking part in another study involving luspatercept. You also need to be willing to come to all the study appointments and follow the study's instructions.

There are a few main ways you might fit in. Firstly, if you're currently receiving luspatercept in another study and your doctor feels it’s still helping you, you might be able to continue on it in this new study. Secondly, if you were on a dummy treatment (placebo) in your previous study and it's decided that you could benefit from luspatercept, you might switch to receiving the actual medicine. Finally, if you've already finished treatment in a previous luspatercept study but are still having your health checked, you might join this study to continue those long-term health checks.

Quick self-check

Are you 18 years old or older?
Are you currently taking part in another study for luspatercept, or have you recently finished one?
Is your doctor recommending you continue with luspatercept, start it, or continue with long-term follow-up after your previous study?
Are you able and willing to attend all study appointments and follow instructions?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, your journey will have different stages. There's an initial 'Transition Phase' which involves one initial check-up visit. Depending on your situation from your previous study, you will then either enter a 'Treatment Phase' or a 'Follow-up Phase'.

If you enter the 'Treatment Phase', you will continue to receive luspatercept at the same dose and schedule you were on in your previous study. After your last dose of luspatercept, you will have a '42 Day Safety Follow-up Visit' where doctors will check your health and any side effects. The 'Follow-up Phase' is much longer; doctors will keep in touch every 6 months for at least 5 years from your very first dose of luspatercept (in your original study), or for 3 years after your last dose, whichever is longer. During this long-term follow-up, they will monitor your general health and look for any new health conditions. The study will continue until all participants have completed 5 years of being in the study, including both treatment and follow-up.

Potential risks and benefits

The main benefit of taking part in this study is that if luspatercept is helping you, you may be able to continue receiving the treatment for longer under careful medical supervision. For others, it provides ongoing health monitoring. As with any medicine, there's always a chance of side effects, and these will be closely watched by the study team. You will have regular check-ups to monitor your health. Remember, you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (143)

Local Institution - 968
Los Angeles, United States· Completed
Local Institution - 971
Oakland, United States· Completed
Local Institution - 978
Stanford, United States· Completed
Local Institution - 975
Tampa, United States· Completed
Local Institution - 970
Chicago, United States· Completed
Local Institution - 973
Boston, United States· Completed
Local Institution - 961
Detroit, United States· Completed
Local Institution - 969
New York, United States· Completed
Local Institution - 967
Cleveland, United States· Completed
Local Institution - 972
Philadelphia, United States· Completed
Vanderbilt - Ingram Cancer Center
Nashville, United States· Withdrawn
The University of Texas - MD Anderson Cancer Center
Houston, United States· Withdrawn

+131 more sites — see the official record for the full list.

Common questions

What is the main purpose of this study?

The main purpose is to evaluate the long-term safety of the medicine luspatercept in people who have already been part of another study for this treatment.

Who can take part in this study?

Adults aged 18 or over who are currently in or have recently completed another clinical trial for luspatercept and meet specific health criteria, as determined by their doctor.

What kind of conditions is luspatercept used for?

Luspatercept is being studied for blood conditions like Myelodysplastic Syndromes (MDS), Beta-thalassemia, and Myelofibrosis.

How long will I be in this study?

This study involves long-term follow-up, which could be for at least 5 years from your first dose of luspatercept or 3 years after your last dose, whichever is longer.

What if I decide I don't want to continue in the study?

You are free to leave the study at any time, for any reason. Your decision will not affect your regular medical care.

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.