Study on Luspatercept for Treating Anemia in Adults with Myelofibrosis on JAK2 Inhibitor Therapy Requiring Red Blood Cell Transfusions

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What is this study about?

This clinical trial is focused on studying a condition called anemia that is associated with a type of blood disorder known as myeloproliferative neoplasm-associated myelofibrosis. This condition often requires patients to receive regular red blood cell transfusions to manage their symptoms. The study is testing a treatment called luspatercept, also known by its code name ACE-536 (BMS-986346), to see if it can help reduce the need for these transfusions. Luspatercept is given as a solution for injection under the skin.

The purpose of the study is to compare the effectiveness and safety of luspatercept with a placebo in adults who are already receiving a type of medication called a JAK2 inhibitor as part of their standard treatment. Participants in the study will receive either luspatercept or a placebo, and their progress will be monitored over a period of time to see if they can become free from needing red blood cell transfusions for at least 12 weeks.

Throughout the study, participants will continue their usual treatment with the JAK2 inhibitor while receiving the study medication. The trial aims to provide valuable information on whether luspatercept can offer a significant benefit in managing anemia in patients with myelofibrosis, potentially improving their quality of life by reducing the frequency of blood transfusions needed.

1 joining the study

Upon joining the study, the participant will be randomly assigned to receive either the study medication, luspatercept, or a placebo. This process is double-blind, meaning neither the participant nor the study team will know which treatment is being administered.

2 treatment administration

The study involves the administration of luspatercept or placebo through subcutaneous injections. The specific products used are Reblozyl 75 mg and Reblozyl 25 mg, both in powder form for solution for injection.

Injections are given at regular intervals as determined by the study protocol. The exact dosage and frequency will be communicated by the study team.

3 monitoring and assessments

Throughout the study, regular monitoring and assessments will be conducted to evaluate the participant’s response to the treatment. This includes tracking the need for red blood cell transfusions and any changes in anemia symptoms.

Participants will undergo various tests and assessments to ensure their safety and to measure the effectiveness of the treatment.

4 blinded core treatment period

The blinded core treatment period lasts up to 24 weeks. During this time, the primary goal is to determine if the participant becomes free from the need for red blood cell transfusions over any consecutive 12-week period.

A secondary goal is to assess if the participant remains transfusion-free over any consecutive 16-week period starting during this treatment phase.

5 end of study participation

At the end of the study, participants will undergo final assessments to evaluate the overall impact of the treatment.

Participants will be informed about the results of the study and their specific treatment allocation (whether they received luspatercept or placebo).

Who Can Join the Study?

The person must be at least 18 years old when they sign the consent form.
The person must have a diagnosis of primary myelofibrosis (PMF) or myelofibrosis that developed after other conditions, confirmed by a recent pathology report. Myelofibrosis is a condition where scar tissue forms in the bone marrow, affecting blood cell production.
The person must need regular red blood cell (RBC) transfusions. This means they need 4 to 12 units of RBCs over 12 weeks, with no gap longer than 6 weeks without at least one transfusion. Transfusions are needed for anemia, which is a low level of red blood cells, causing symptoms like tiredness or shortness of breath.
The person must be on continuous JAK2 inhibitor therapy, a type of medication for myelofibrosis, for at least 32 weeks, with a stable dose for at least 16 weeks before joining the study. They should also be expected to continue this stable dose for at least 24 weeks after joining the study.
The person must have an Eastern Cooperative Oncology Group (ECOG) performance score of 2 or less. This score measures how well a person can perform daily activities, with lower scores indicating better ability.
Females who can have children must have two negative pregnancy tests before starting the study and agree to ongoing pregnancy testing. They must either practice true abstinence from heterosexual contact or use effective birth control before and during the study, and for 12 weeks after stopping the study treatment.
Male participants must practice true abstinence or use a condom during sexual contact with a pregnant female or a female who can have children while in the study, during breaks in treatment, and for at least 12 weeks after stopping the study treatment, even if they have had a vasectomy.

Who Cannot Join the Study?

Patients who do not have anemia related to a condition called myeloproliferative neoplasm-associated myelofibrosis cannot participate. This is a type of blood disorder.
Patients who are not currently receiving treatment with a JAK2 inhibitor cannot participate. JAK2 inhibitors are medications used to treat certain blood disorders.
Patients who do not need regular red blood cell transfusions cannot participate. A red blood cell transfusion is a procedure where you receive blood through a vein.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
University Hospital Jena KöR	Jena	Germany
Hospital Universitario De Salamanca	Salamanca	Spain
Centre Hospitalier Universitaire De Lille	Lille	France
Katholieke Universiteit te Leuven	Leuven	Belgium
University Hospital Of Clermont-Ferrand	Clermont Ferrand	France
CHU Grenoble Alpes	La Tronche	France

Other Sites

Site Name	City	Country
Del-Pesti Centrumkorhaz Orszagos Hematologiai Es Infektologiai Intezet	Budapest	Hungary
Centre Hospitalier Universitaire De Nice	Nice	France
Specjalistyczny Szpital Im. Dra Alfreda Sokolowskiego	Walbrzych	Poland
Institute Of Oncology Prof. Dr. Ion Chiricuta Cluj-Napoca	Cluj Napoca	Romania
Aockevx Oqjmpnyjphw dx Pzjuyr	Padua	Italy
Egahynbxrhit Srqo	Athens	Greece
Vsvywu dai Rpgbn Uaohigiebo Hvtyhwvz	Sevilla	Spain
Ftjhrvhkem Ixbbp Cm Gguldo Ozikcgbl Mdycptkp Pbekhfmmrch	Milan	Italy
Iailtqdd Rzocemgww Peo Ls Sawrgb Dqe Tloilq Dpdd Ahhxpkz Iyul Seeseb	Meldola	Italy
Hxlulshv Cdatxa Dn Bfxdbrbfa	Barcelona	Spain
Ugzfppdjdyhnhebca Pchgxiplfiy Deqdu Myqylt	Ancona	Italy
Ubqnruntqeqj Dk Spdmmcnw Dp Cgglagpvhz	Santiago De Compostela	Spain
Sspqlwpumeh Prjbbjhdq Zccbxe Oouosb Zurchvakad Srhrrhb Uyqvunlsrpnrx W Kqhwajag	Cracow	Poland
Asmvhrt Oezqdqteurf Ufebnqiubwgiw Cbykhughredt Dqpry Sooecu E Dwieb Scacwjj Dh Txicoj	Turin	Italy
Atmrzvo Obclkvjmbse Unnriqgfzcnfb Fyrewiyw Is Dq Ncucfl	Naples	Italy
Gpwifqb Ukkcjbjaqi Huhixxdq Oy Pqrgzo	Patras	Greece
Urhosqnxoe Grnqufz Hxnoquth Of Ambyeruwsfxumm	Alexandroupoli	Greece
Aikmbto Oayqwdzhbsxaivmzvilkesryu Masqxlax Duugc Cgfpmf	Novara	Italy
Vvfzgeyde Fvgpxzhm Nmbdbwwkm V Pigiy	Prague	Czechia
Cptixk Lzky Bjwtvl	Lyon	France
Cozabz Hqvpizessra Ucxsytqgtcmky Dq Nptrh	Nimes	France
Onqjvwgqfnnyzx Lyfe Gydp	Linz	Austria
Ctagru hqkipcqtmrj ubepeabfvxcqz da Lexqs	Liege	Belgium
Hulzaqrm Uwddjcsggqjty 1w Dh Ohpeaot	Madrid	Spain
Uaspehlqlw Dxphe Sqbbg Du Biotofu	Brescia	Italy
Cqfcne Hyddeheymhc Rictyyvv Dnafgceyquamtc	Angers	France
Ghyzxz Ngxmecyuvi Tnspyexdwwpti Gicstf Pdpidwvbohbc	Thessaloniki	Greece
Creuoi Hmrcakxardc Utmnoeahejqlm Dpicyv Gcrhhng Saknzdtqihyzbiwkutvxfktxla	Namur	Belgium
Jqgdezqc Weujpcy Krggzdwt Meicbt	Minden	Germany
Hviqrybt Ufvpfqptpigla Vqlxok Dm Lvm Nkgyba	Granada	Spain
Adrhuva Sudjg Sgywhpbgq Turdjitllgny Drg Sjzgu Lnlpr	Varese	Italy
Hytphwsn Ubuqgldwfkxbdd Scndcojrex &xfjuup Hzhrgfm dg Hyyxltuegdb	STRASBOURG, Alsace	France
Ulxpklsueb Gtdfnwf Hxleyjbh Aacfdwm Glfhxxr Htnkqkqo Om Woyd Atvxgu H Apnw Vflhoil	Chaidari	Greece
Aulmfce Oayzqfpzzodlnkloxucawtmkp Pixbqtswrom Gj Rcqjyvgiqfhr Miikg Dt Cqzgfpe	Catania	Italy
Skgpxdft Cbkjng Mudfztuly Frrxishngit Cgzhlkq	Craiova	Romania
Oxfucmvh Sa Erzqprg	Rome	Italy
Agnnejhukk Pyanwnst Habwkfjm Dx Piane	Paris	France
Gubtshqgmuqsbixrs Vehxqhwvx Pxdw Abkedy Epevlehg Onynaq Kuhozp	Gyor	Hungary
Ovdj Cfoj Sgckdq	Brasov	Romania
Csckri Rwjfuafy Chsfxora Dj Vehmjv Sftefk	Verona	Italy
Csnffo Hdolybirjgh Ucebcpjuubahn Dq Pvrlktxp	Poitiers	France
Mopugegispdjceyjwartocnxvr Hnlghkyxxibqjixz	Halle (Saale)	Germany
Cneb Urhxtwqduq Hriuwaje	Cork	Ireland
Clg Vxleivpk	Verviers	Belgium
Et Hgjxfqss Uadnouxuwmrpq Dg Gekz Cerhoce Dnn Njjjgi	Las Palmas De Gran Canaria	Spain
Hstmpjtl Uazkrmrviwwfd Hhhkbplq Tzdnw y Piiqqa Iunvdecp Cofbte dnnhajzhlntxyyrln (dtag	Badalona	Spain
Oyeaqnlw Tkzkyxtvzvuek Geqtvbx Cgiyfr Oi Pbtniu Ssnl	Patras	Greece

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	30.01.2021
Belgium	Not recruiting	30.01.2021
Czechia	Not recruiting	30.01.2021
France	Not recruiting	30.01.2021
Germany	Not recruiting	30.01.2021
Greece	Not recruiting	30.01.2021
Hungary	Not recruiting	30.01.2021
Ireland	Not recruiting	30.01.2021
Italy	Not recruiting	30.01.2021
Poland	Not recruiting	30.01.2021
Romania	Not recruiting	30.01.2021
Spain	Not recruiting	30.01.2021

Trial locations

Investigated Drugs:

Luspatercept (ACE-536) is a medication being studied for its ability to treat anemia in patients with myeloproliferative neoplasm-associated myelofibrosis. This condition often requires patients to receive red blood cell transfusions. Luspatercept works by helping the body produce more red blood cells, potentially reducing the need for transfusions. It is being tested in combination with JAK2 inhibitor therapy, which is commonly used to manage symptoms of myelofibrosis.

Investigated Diseases:

Anemia associated with myeloproliferative neoplasm-associated myelofibrosis – This condition occurs when the bone marrow, which is responsible for producing blood cells, becomes scarred and fibrotic, leading to a decrease in the production of red blood cells. As a result, individuals experience anemia, characterized by fatigue, weakness, and pallor due to insufficient oxygen being transported throughout the body. Myeloproliferative neoplasms are a group of diseases where the bone marrow makes too many blood cells, and when associated with myelofibrosis, it can lead to further complications in blood cell production. The progression of this condition involves worsening anemia and may require regular blood transfusions to manage symptoms. Over time, the bone marrow’s ability to produce healthy blood cells continues to decline, exacerbating the symptoms of anemia.

Trial ID:

2023-507890-17-00

Protocol code:

ACE-536-MF-002

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Luspatercept for Treating Anemia in Adults with Myelofibrosis on JAK2 Inhibitor Therapy Requiring Red Blood Cell Transfusions

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?