Clinical trial NCT06045689: A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants
Official title: A Phase 3b, Open-label Study Evaluating the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) Initiated at Maximum Approved Dose in LR-MDS With IPSS-R Very Low-, Low-, or Intermediate-risk Who Require RBC Transfusions (MAXILUS)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Myelodysplastic Syndromes
Keywords: Luspatercept
Transfusion dependent
ACE-536
Anemia
Blood Transfusion
Red Blood Cell Transfusion
Myelodysplastic Syndrome
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
First posted: 2023-09-21
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the efficacy and safety of Luspatercept when administered at the maximum approved dose in low-risk Myelodysplastic Syndrome participants who require red blood cell transfusions.


Eligibility criteria: Inclusion Criteria:

* Participant had documented diagnosis of MDS according to World Health Organization (WHO) classification that met Revised International Prognostic Scoring System (IPSS-R) classification of very low-, low-, or intermediate-risk disease.
* Participant has an Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2.
* Participant must have red blood cell transfusions according to study criteria.

Exclusion Criteria:

* Participant has known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding.
* Participant has had a prior allogeneic or autologous stem cell transplant.
* Participant has known history or diagnosis of AML.
* Participant has uncontrolled hypertension.

Other protocol-defined inclusion/exclusion criteria apply

Primary outcomes:
measure: Number of participants who achieve red blood cell transfusion independence (RBC-TI) for 8 weeks with a simultaneous mean hemoglobin (Hb) increase of ≥ 1 g/dL from Week 1 to Week 24
timeFrame: Up to week 24
Secondary outcomes:
measure: Number of participants with a mean change in total RBC units transfused over a fixed 16-week period from Week 9 to Week 24 and from Week 33 to Week 48
timeFrame: Up to week 48
measure: Number of participants who have a time from first dose to first onset of RBC-TI ≥ 8-, 12-, and 16-weeks from Week 1 to Week 24, from Week 1 to Week 48, and from Week 1 through EOT
timeFrame: Up to 2 years
measure: Number of participants who achieve RBC-TI over any consecutive 8-week period from Week 1 to Week 24, from Week 1 to Week 48, and from Week 1 to EOT
timeFrame: Up to 2 years
measure: Number of participants with a maximum duration of RBC-TI for participants who achieve RBC TI ≥ 8- and 16-week period from Week 1 to Week 24 and from Week 1 to EOT
timeFrame: Up to 2 years
measure: Number of participants who achieve RBC-TI over any consecutive 12-, 16-, and 24-week periods from Week 1 to Week 24, from Week 1 to Week 48 and from Week 1 through EOT
timeFrame: Up to 2 years
measure: Number of participants with an increase from baseline in mean hemoglobin (Hb) values of ≥ 1.0 g/dL over any consecutive 8-week period in absence of RBC transfusions from Week 1 to Week 48 and from Week 1 through EOT
timeFrame: Up to 2 years
measure: Number of participants with an increase from baseline in Hb values of ≥ 1.0 g/dL over any consecutive 16-week period in absence of RBC transfusions from Week 1 to Week 24, from Week 1 to Week 48, and from Week 1 through EOT
timeFrame: Up to 2 years
measure: Number of participants with an increase from baseline in Hb values of ≥ 1.5 g/dL over any consecutive 8-, and 16-week periods in absence of RBC transfusions from Week 1 to Week 24, from Week 1 to Week 48, and from Week 1 through EOT
timeFrame: Up to 2 years
measure: Number of participants who achieve Hematological Improvement Erythroid (mHI-E) per International Working Group-2018 (IWG-2018) over any consecutive 8-week period from Week 1 to Week 24, from Week 1 to Week 48, and Week 1 through EOT
timeFrame: Up to 2 years
measure: Number of participants who achieve Hematological Improvement - Neutrophils (HI-N) per IWG-2018 over any consecutive 8-week period from Week 1 to Week 24, from Week 1 to Week 48, and Week 1 through EOT
timeFrame: Up to 2 years
measure: Number of participants who achieve Hematological Improvement - Platelets (HI-P) per IWG-2018 over any consecutive 8-week period from Week 1 to Week 24, from Week 1 to Week 48, and Week 1 through EOT
timeFrame: Up to 2 years
measure: Number of participants with change in serum ferritin (SF) over a 16-week period from Week 9 to Week 24 and from Week 33 to Week 48
timeFrame: Up to week 48
measure: Number of participants with change in mean daily dose of iron chelation therapy (ICT) over a 16-week period from Week 9 to Week 24 and from Week 33 to Week 48
timeFrame: Up to week 48
measure: Number of participants with adverse events (AEs)
timeFrame: Up to 2 years
measure: Number of participants with acute myeloid leukemia (AML) progression
timeFrame: Up to 4 years
measure: Time to AML progression
timeFrame: Up to 4 years
measure: Time from treatment start date to death due to any cause
timeFrame: Up to 4 years
measure: Number of participants with a change in subscale scores of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) from Week 1 to Week 48 and from baseline through EOT
timeFrame: Up to 2 years
Other outcomes:
Locations:
city: Los Alamitos
country: United States
facility: Cancer and Blood Specialty Clinic
state: California
city: San Diego
country: United States
facility: Local Institution - 0048
state: California
city: New Haven
country: United States
facility: Smilow Cancer Hospital at Yale New Haven
state: Connecticut
city: Miami
country: United States
facility: Local Institution - 0042
state: Florida
city: Saint Petersburg
country: United States
facility: Florida Cancer Specialists - NORTH - SCRI - PPDS
state: Florida
city: Wellington
country: United States
facility: Florida Cancer Specialists - SOUTH - SCRI - PPDS
state: Florida
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Paducah
country: United States
facility: Mercy Health - Paducah Medical Oncology and Hematology
state: Kentucky
city: Worcester
country: United States
facility: Local Institution - 0012
state: Massachusetts
city: Detroit
country: United States
facility: Henry Ford Hospital
state: Michigan
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: Morristown
country: United States
facility: Atlantic Hematology Oncology
state: New Jersey
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
city: Durham
country: United States
facility: Local Institution - 0057
state: North Carolina
city: Cleveland
country: United States
facility: Local Institution - 0060
state: Ohio
city: Columbus
country: United States
facility: Local Institution - 0006
state: Ohio
city: Oklahoma City
country: United States
facility: Local Institution - 0061
state: Oklahoma
city: Eugene
country: United States
facility: Oncology Associates of Oregon, P.C.
state: Oregon
city: Pittsburgh
country: United States
facility: West Penn Hospital
state: Pennsylvania
city: Amarillo
country: United States
facility: Texas Oncology - Amarillo
state: Texas
city: Huntsville
country: United States
facility: Local Institution - 0022
state: Texas
city: Wheeling
country: United States
facility: Wheeling Hospital Schiffler Cancer Center
state: West Virginia
city: Milwaukee
country: United States
facility: Froedtert and The Medical College of Wisconsin
state: Wisconsin
city: Leuven
country: Belgium
facility: UZ Leuven- Gasthuisberg Campus
state: Vlaams Brabant
city: Roeselare
country: Belgium
facility: AZ Delta- Campus Rumbeke
state: West-Vlaanderen
city: Brno
country: Czechia
facility: Local Institution - 0064
state: Jihomoravský Kraj
city: Praha 2
country: Czechia
facility: Vseobecna Fakultni Nemocnice V Praze-U Nemocnice 499/2
state: Praha, Hlavní Mesto
city: Praha
country: Czechia
facility: Ustav hematologie a krevni transfuze
state: Praha, Hlavní Mesto
city: Nice
country: France
facility: CHU de Nice Archet I
state: Alpes-Maritimes
city: Poitiers
country: France
facility: CHU de Poitiers
state: Vienne
city: Angers
country: France
facility: CHU d'Angers
city: Grenoble cedex 09
country: France
facility: CHU de Grenoble Alpes - Hôpital Michallon
city: Paris
country: France
facility: AP-HP - Hôpital Saint-Louis
city: Pierre-Bénite
country: France
facility: Hospices Civils de Lyon - Hôpital Lyon Sud
city: Tour Cedex01
country: France
facility: Hôpital Bretonneau
city: München
country: Germany
facility: Klinikum rechts der Isar der Technischen Universitaet Muenchen
state: Bayern
city: Hannover
country: Germany
facility: Studienzentrum am Raschplatz GbR
state: Niedersachsen
city: Gütersloh
country: Germany
facility: Local Institution - 0037
state: Nordrhein-Westfalen
city: Leipzig
country: Germany
facility: Universitatsklinikum Leipzig
state: Sachsen
city: Reggio Calabria
country: Italy
facility: Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli - PO Riuniti
state: Calabria
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II
state: Campania
city: Roma
country: Italy
facility: Fondazione PTV Policlinico Tor Vergata
state: Lazio
city: Pavia
country: Italy
facility: Fondazione IRCCS Policlinico San Matteo
state: Lombardia
city: Rozzano (MI)
country: Italy
facility: IRCCS Istituto Clinico Humanitas
state: Milano
city: Novara
country: Italy
facility: Azienda Ospedaliero Universitaria Maggiore della Carità
state: Piemonte
city: Torino
country: Italy
facility: Azienda Ospedaliera Ordine Mauriziano di Torino
state: Piemonte
city: Firenze
country: Italy
facility: Azienda Ospedaliera Universitaria Careggi
state: Toscana
city: Wroclaw
country: Poland
facility: Local Institution - 0002
state: Dolnoslaskie
city: Lublin
country: Poland
facility: Local Institution - 0030
state: Lubelskie
city: Warszawa
country: Poland
facility: MTZ Clinical Research Powered by PRATIA - PPDS
state: Mazowieckie
city: Katowice
country: Poland
facility: Pratia Onkologia Katowice - PRATIA - PPDS
city: Lodz
country: Poland
facility: Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi
city: Wałbrzych
country: Poland
facility: Specjalistyczny Szpital im. Alfreda Sokolowskiego
city: San Juan
country: Puerto Rico
facility: Auxilio Mutuo Cancer Center
city: L'Hospitalet de Llobregat
country: Spain
facility: ICO l'Hospitalet - Hospital Duran i Reynals
state: Barcelona
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron - PPDS
city: Barcelona
country: Spain
facility: Hospital Universitario Germans Trias i Pujol
city: Granada
country: Spain
facility: Hospital Universitario Virgen de Las Nieves
city: Madrid
country: Spain
facility: Hospital Universitario La Princesa
city: Ourense
country: Spain
facility: CHU de Ourense - H. Santa Maria Nai
city: Salamanca
country: Spain
facility: Complejo Asistencial Universitario de Salamanca - H. Clinico
city: Valencia
country: Spain
facility: Hospital Clinico Universitario de Valencia
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe de Valencia
Study officials:
affiliation: Bristol-Myers Squibb
name: Bristol-Myers Squibb
role: STUDY_DIRECTOR
References: