Study on Long-Term Safety of Luspatercept for Patients with Myelodysplastic Syndrome, Beta-Thalassemia, or Myelofibrosis Who Previously Participated in Luspatercept Trials

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What is this study about?

This clinical trial is focused on studying the long-term safety of a treatment called luspatercept, also known by its code name ACE-536 or BMS-986346. The study involves participants who have previously taken part in other trials using this treatment. The diseases being studied include myelodysplastic syndrome (MDS), beta-thalassemia, and myelofibrosis. These are conditions that affect the blood and bone marrow, leading to issues like anemia and other blood-related problems.

The purpose of this study is to evaluate the long-term safety of luspatercept, including monitoring for any progression to more serious conditions such as acute myeloid leukemia (AML) or the development of other types of cancer. Participants in this study will receive luspatercept as a solution for injection, which is administered under the skin. The study is designed as an open-label, single-arm, rollover study, meaning all participants will receive the treatment without a comparison group receiving a placebo.

Throughout the study, participants will be monitored for any adverse events or changes in their condition. The study will also track overall survival and any new growths that may develop. The maximum treatment period for participants is up to 360 days, during which they will continue to receive the treatment and be regularly assessed by healthcare professionals. This study aims to provide valuable information on the long-term effects and safety of luspatercept for individuals with these blood disorders.

1 Enrollment

Upon joining the study, eligibility is confirmed. This includes being 18 years or older and having participated in a previous luspatercept trial.

An informed consent document is signed, indicating understanding and agreement to the study’s requirements.

2 Initial Assessment

Initial assessments are conducted to establish a baseline for health and safety monitoring.

For females of childbearing potential, two negative pregnancy tests are required before starting the study therapy.

3 Treatment Phase

The treatment involves receiving luspatercept through subcutaneous injections.

The medication is available in two forms: Reblozyl 25 mg and Reblozyl 75 mg powder for solution for injection.

The frequency and dosage are determined by the study protocol and the investigator’s assessment of clinical benefit.

4 Monitoring and Follow-up

Regular monitoring is conducted to evaluate long-term safety, including any adverse events or progression to other conditions.

Participants are required to adhere to the study visit schedule for ongoing assessments.

5 End of Study

The study is estimated to conclude by February 23, 2029.

Upon completion, participants may undergo final assessments to evaluate overall health and any long-term effects of the treatment.

Who Can Join the Study?

The person must be at least 18 years old when they sign the consent form.
The person must be willing and able to follow the schedule of study visits and other study requirements.
The person must already be part of a study involving a treatment called luspatercept and continue to meet the requirements of that study. They must be in one of the following situations:
- Currently receiving luspatercept and benefiting from it, according to the study doctor.
- Previously received a placebo (a harmless pill with no effect) and should now start luspatercept treatment.
- In the follow-up phase of the previous study, having been treated with either luspatercept or placebo, and should continue in the long-term follow-up phase.
The person must understand and willingly sign a consent form before any study-related tests or procedures are done.
The person must have followed the rules of the previous study, as checked by the study doctor.
For women who can have children and are currently receiving treatment:
- They must have two negative pregnancy tests before starting the study treatment.
- They must agree to regular pregnancy tests during and after the study.
- They must either commit to not having sexual contact or agree to use effective birth control methods before, during, and after the study.
For men currently receiving treatment:
- They must agree to use a condom during sexual contact with a pregnant woman or a woman who can have children while in the study and for a period after stopping the study treatment, even if they have had a vasectomy (a procedure to prevent pregnancy).

Who Cannot Join the Study?

Patients who have not participated in other clinical trials involving the medication called luspatercept.
Patients who have a history of progressing to a condition called acute myeloid leukemia (AML), which is a type of blood cancer.
Patients who have developed other types of cancers or pre-cancerous conditions.
Patients who are not within the specified age range for the study.
Patients who are not part of the specified clinical trial groups.
Patients who are not male or female, as the study includes both genders.
Patients who are considered part of a vulnerable population, which means they might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Azienda Ospedaliera Universitaria Universita’ Degli Studi Della Campania Luigi Vanvitelli	Naples	Italy
Center For Pediatric And Adolescent Medicine Of The Johannes Gutenberg University Mainz	Mainz	Germany
Hospital Universitario De Salamanca	Salamanca	Spain
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy
Technische Universitaet Dresden	Dresden	Germany

Other Sites

Site Name	City	Country
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)	Munich	Germany
Nosokomeio Paidon I Agia Sofia	Athens	Greece
Ippokratio General Hospital Of Thessaloniki	Thessaloniki	Greece
Grande Ospedale Metropolitano Bianchi Melacrino Morelli	Reggio Calabria	Italy
University Multiprofile Hospital For Active Treatment Saint Georgi EAD	Plovdiv	Bulgaria
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Azienda Ospedaliero-Universitaria San Luigi Gonzaga	Orbassano	Italy
Ente Ospedaliero Ospedali Galliera Di Genova	Genoa	Italy
Hopital Beaujon	Clichy	France
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen	Gothenburg	Sweden
Specialized Hospital For Active Treatment Of Hematological Diseases EAD	Sofia	Bulgaria
Lsdvg Gofkyij Hjphgucd Ov Ajbpee	Athens	Greece
Afabrencg Uph	Amsterdam	The Netherlands
Mjjititxhmmidgxtdzggrxlrlm Hurmhloazcyzmbav	Halle (Saale)	Germany

Trial status

Country	Status	Recruitment Start
Bulgaria	Not recruiting	01.07.2019
France	Not recruiting	01.07.2019
Germany	Not recruiting	01.07.2019
Greece	Recruiting	01.07.2019
Italy	Recruiting	01.07.2019
Spain	Not recruiting	01.07.2019
Sweden	Not recruiting	01.07.2019
The Netherlands	Not recruiting	01.07.2019

Trial locations

Investigated drugs:

Luspatercept

Luspatercept is a medication being studied for its long-term safety in patients who have previously participated in other clinical trials involving this drug. It is used to treat certain types of anemia by helping the body produce more red blood cells. This study aims to monitor any potential progression to acute myeloid leukemia or other related conditions in patients using luspatercept over an extended period.

Investigated diseases:

Myelodysplastic Syndrome (MDS) – This is a group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when the blood-forming cells in the bone marrow are damaged, leading to a shortage of one or more types of blood cells. Over time, MDS can progress to more severe forms, affecting the production of red blood cells, white blood cells, and platelets. Patients may experience symptoms like fatigue, infections, or easy bruising due to low blood counts. The condition can vary in severity, with some cases remaining stable for years while others progress more rapidly.

Beta-Thalassemia (THAL) – This is a blood disorder that reduces the production of hemoglobin, the protein in red blood cells that carries oxygen. It is caused by mutations in the HBB gene, leading to anemia and fatigue. The severity of beta-thalassemia can range from mild to severe, depending on the specific genetic mutations. In severe cases, individuals may require regular blood transfusions to manage symptoms. Over time, the condition can lead to complications such as bone deformities and growth delays.

Myelofibrosis (MF) – This is a rare type of bone marrow cancer that disrupts the body’s normal production of blood cells. It leads to extensive scarring in the bone marrow, resulting in severe anemia, weakness, and fatigue. As the disease progresses, it can cause an enlarged spleen and liver, as well as other complications. Patients may experience symptoms such as night sweats, fever, and bone pain. The progression of myelofibrosis can vary, with some individuals experiencing a slow course while others may have a more rapid decline.

Trial ID:

2022-502498-40-00

Protocol code:

ACE-536-LTFU-001

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Long-Term Safety of Luspatercept for Patients with Myelodysplastic Syndrome, Beta-Thalassemia, or Myelofibrosis Who Previously Participated in Luspatercept Trials

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?