Study on Luspatercept and Epoetin Alfa for Patients with Low-Risk Myelodysplastic Syndrome Without Ring Sideroblasts Who Did Not Respond to ESA

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What is this study about?

This clinical trial is focused on studying a condition known as low-risk myelodysplastic syndrome (MDS), which is a type of blood disorder where the bone marrow does not produce enough healthy blood cells. The study is investigating the effectiveness of a treatment combination involving two medications: luspatercept and epoetin alfa. Luspatercept is a medication that helps improve the production of red blood cells, while epoetin alfa is used to stimulate the production of red blood cells in the bone marrow.

The purpose of the study is to determine the best dose of luspatercept when used in combination with epoetin alfa and to evaluate whether this combination is more effective than using luspatercept alone. The study is divided into two parts. In the first part, researchers will find the optimal dose of the combination treatment by observing its effects on patients. In the second part, they will compare the benefits of the combination treatment to luspatercept alone, focusing on improvements in blood cell counts and the need for blood transfusions.

Participants in the study will receive the treatments through injections under the skin. The study will monitor the participants over a period of time to assess the treatment’s impact on their condition, including any changes in their need for blood transfusions and overall health improvements. The study aims to provide valuable information on how these treatments can help manage low-risk myelodysplastic syndrome more effectively.

1 joining the trial

Upon joining the trial, the patient will be required to sign a consent form, confirming understanding and voluntary participation in the study.

The patient must adhere to the visit schedule and follow all protocol requirements as outlined in the study.

2 initial assessment

The patient will undergo an initial assessment to confirm eligibility, which includes a review of medical history and current health status.

Eligibility criteria include having a low-risk myelodysplastic syndrome without RS, having failed or being ineligible for erythroid stimulating agents.

3 treatment phase part A

In Part A, the patient will receive a combination of luspatercept and epoetin alfa through subcutaneous injections. The exact dosage will be determined during this phase.

The goal is to find the optimal dose that balances effectiveness and tolerability. The observation period lasts up to day 42 of cycle 1.

4 treatment phase part B

In Part B, the patient will continue to receive the combination treatment or luspatercept alone, depending on the study’s randomization process.

The effectiveness of the treatment will be evaluated at week 25, focusing on achieving transfusion independence or hematological improvement.

5 monitoring and follow-up

Throughout the trial, the patient will have regular check-ups to monitor health status and treatment response.

The patient will be required to undergo periodic blood tests to assess hemoglobin levels and other relevant health indicators.

6 end of treatment

At the end of the treatment phase, the patient will have a final assessment to evaluate the overall response to the treatment.

The patient may be asked to continue follow-up visits to monitor long-term health outcomes and any potential side effects.

Who Can Join the Study?

Must have a condition called myelodysplastic syndrome as per the current WHO classification.
Must understand and voluntarily sign a consent form.
Must be able to follow the visit schedule and study requirements.
Must have an ECOG performance status of 0-2 at the time of screening. (This is a scale used to assess how a disease affects a patient’s daily living abilities.)
Must be 18 years or older.
Must have lower risk myelodysplastic syndrome according to the IPSS classification without RS, and have not responded to or relapsed after treatment with an Erythroid Stimulating Agent (ESA), or be ineligible for ESA.
Must have a hemoglobin level of less than 9 grams per deciliter or be dependent on blood transfusions (at least 3 red blood cell transfusions in 16 weeks in at least 2 separate episodes).
Must not have a condition called del(5q) syndrome.
Must have adequate kidney function, defined by a creatinine level less than 1.5 times the upper limit of normal and a creatinine clearance of at least 40 mL/min.
Must have adequate liver function, defined by total bilirubin and transaminases levels less than 1.5 times the upper limit of normal.
Must not be known to be resistant to platelet transfusions.
Must provide written informed consent.
If female and of childbearing potential, must have two negative pregnancy tests before starting the study and agree to ongoing monthly pregnancy testing during the study and after its end.
If sexually active, must agree to use highly effective contraception methods before, during, and after the study.
Male participants must agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study and for at least 12 weeks after stopping the study treatment.

Who Cannot Join the Study?

Patients with a different type of myelodysplastic syndrome (MDS) than the one specified in the study.
Patients who have not failed or are not ineligible for treatment with an Erythroid Stimulating Agent (ESA). An ESA is a medication that helps the body make more red blood cells.
Patients who have shown disease progression, meaning their condition has worsened over time.
Patients who are not within the specified age range for the study.
Patients who are not part of the specified clinical trial group.
Patients who are not considered part of the vulnerable population selected for the study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Centre Hospitalier Universitaire De Lille	Lille	France
Oncopole Claudius Regaud	Toulouse	France
CHU Grenoble Alpes	La Tronche	France
Azienda Ospedaliero Universitaria Careggi	Florence	Italy

Other Sites

Site Name	City	Country
Centre Hospitalier De Versailles	Le Chesnay-Rocquencourt	France
Centre Hospitalier Victor Dupouy	Argenteuil	France
Centre Hospitalier Universitaire De Nimes	Nimes	France
Centre Hospitalier Universitaire De Poitiers	Poitiers	France
Centre Hospitalier D Avignon	Avignon	France
Centre Henri Becquerel	Rouen	France
Clinique de l’Europe	Amiens	France
Centre Hospitalier Universitaire d’Orléans	Orléans	France
Istituto Di Candiolo Fondazione Del Piemonte Per Loncologia IRCCS	Candiolo	Italy
Centre Hospitalier Le Mans	Le Mans	France
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Centre Hospitalier De La Cote Basque	Bayonne	France
Centre Hospitalier Universitaire De Nantes	Nantes	France
Hopital NOVO	Pontoise	France
Centre Hospitalier Universitaire De Nice	Nice	France
Centre Hospitalier De Perigueux	Perigueux	France
Centre Hospitalier Intercommunal Compiegne Noyon	Compiegne	France
Centre Hospitalier Universitaire Amiens Picardie	Amiens	France
Centre Hospitalier Universitaire De Rennes	Rennes	France
Centre Hospitalier Lyon Sud	Pierre Benite	France
Hopital Beaujon	Clichy	France
L’Hopital Prive Du Confluent	Nantes	France
Bicetre Hospital	Le Kremlin-Bicetre	France
Centre Hospitalier Intercommunal De Mont De Marsan Et Du Pays Des Sources	Mont-de-Marsan	France
Crogni Hglbpwxwhex Vubojuz	Valence	France
Ccyvsw dv Rsvbraedqtwyo &jnueas Cbmkbnoy Sbffof Abfv	STRASBOURG, Alsace	France
Cbsacc Hmdfzbwtdgv Er Utwwemkoflpqh Da Lujlkqy	Limoges	France
Ckrspg Hcgexsluidt Rdgetzmg Ugmykoqfvzouj Dc Tkxmf	Tours	France
Csdg Df Ntrpt	Vandoeuvre Les Nancy	France
Cssknr Hmcodkqryup Rljbupcg Dfmihzktjvaexu	Angers	France
Iugvjpeq db Chbklkepnoys Hvmelmtwtgw Uperlbjivaisv db Snmpi Eqzeayk (jgvshtk	Saint Priest En Jarez	France

Trial status

Country	Status	Recruitment Start
France	Recruiting	18.05.2022
Italy	Recruiting	18.05.2022

Trial locations

Investigated drugs:

Luspatercept is a medication used in this trial to help improve blood cell production in patients with a specific type of blood disorder called lower-risk myelodysplastic syndromes (MDS). This condition can cause low levels of red blood cells, leading to anemia. Luspatercept works by helping the body produce more red blood cells, which can reduce the need for blood transfusions and improve overall blood health.

Erythropoietin (EPO) is a therapy used in combination with luspatercept in this trial. EPO is a hormone that naturally occurs in the body and stimulates the production of red blood cells. In this trial, EPO is used to enhance the effects of luspatercept, aiming to further increase red blood cell production and improve the patient’s condition by reducing anemia symptoms and the need for blood transfusions.

Myelodysplastic Syndrome – Myelodysplastic Syndrome (MDS) 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 decrease in the number of healthy blood cells. Over time, this can result in symptoms such as fatigue, shortness of breath, and increased risk of bleeding or infection due to low blood counts. The disease progresses as the bone marrow becomes less effective at producing normal blood cells, which can lead to anemia, neutropenia, or thrombocytopenia. In some cases, MDS can evolve into acute myeloid leukemia, a more aggressive form of blood cancer. The progression of MDS varies among individuals, with some experiencing slow changes and others facing more rapid developments.

Trial ID:

2024-515354-24-00

Protocol code:

COMBOLA

NCT ID:

NCT05181735

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on Luspatercept and Epoetin Alfa for Patients with Low-Risk Myelodysplastic Syndrome Without Ring Sideroblasts Who Did Not Respond to ESA

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?