MAXILUS: A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants

Sponsor

Bristol-Myers Squibb (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT06045689

Collaborator

(none)

100

Enrollment

Locations

Arms

50.5

Anticipated Duration (Months)

1.9

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Myelodysplastic Syndromes	Drug: Luspatercept	Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

100 participants

Allocation:

Non-Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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)

Anticipated Study Start Date :

Oct 15, 2023

Anticipated Primary Completion Date :

Jan 1, 2026

Anticipated Study Completion Date :

Dec 30, 2027

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cohort 1: erythropoiesis-stimulating agents (ESA) naïve	Drug: Luspatercept Specified dose on specified days. Other Names: BMS-986346 ACE-536 REBLOZYL
Experimental: Cohort 2: ESA relapsed or refractory	Drug: Luspatercept Specified dose on specified days. Other Names: BMS-986346 ACE-536 REBLOZYL

Arm

Intervention/Treatment

Experimental: Cohort 1: erythropoiesis-stimulating agents (ESA) naïve

Drug: Luspatercept

Specified dose on specified days.

Other Names:

BMS-986346

ACE-536

REBLOZYL

Experimental: Cohort 2: ESA relapsed or refractory

Drug: Luspatercept

Specified dose on specified days.

Other Names:

BMS-986346

ACE-536

REBLOZYL

Outcome Measures

Primary Outcome Measures

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 [Up to week 24]

Secondary Outcome Measures

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 [Up to week 48]
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 [Up to 2 years]
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 [Up to 2 years]
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 [Up to 2 years]
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 [Up to 2 years]
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 [Up to 2 years]
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 [Up to 2 years]
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 [Up to 2 years]
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 [Up to 2 years]
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 [Up to 2 years]
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 [Up to 2 years]
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 [Up to week 48]
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 [Up to week 48]
Number of participants with adverse events (AEs) [Up to 2 years]
Number of participants with acute myeloid leukemia (AML) progression [Up to 4 years]
Time to AML progression [Up to 4 years]
Time from treatment start date to death due to any cause [Up to 4 years]
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 [Up to 2 years]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Cancer and Blood Specialty Clinic	Los Alamitos	California	United States	90720-3379
2	Scripps Prebys Cancer Center	San Diego	California	United States	92103-2106
3	Smilow Cancer Hospital at Yale New Haven	New Haven	Connecticut	United States	06510
4	Sylvester Comprehensive Cancer Center-Miami	Miami	Florida	United States	33136
5	University of Kansas Medical Center	Kansas City	Kansas	United States	66160-8500
6	Mercy Health - Paducah Medical Oncology and Hematology	Paducah	Kentucky	United States	42003-7915
7	UMass Memorial Medical Center	Worcester	Massachusetts	United States	01655
8	Henry Ford Hospital	Detroit	Michigan	United States	48202
9	Dartmouth Hitchcock Medical Center	Lebanon	New Hampshire	United States	03766
10	Icahn School of Medicine at Mount Sinai	New York	New York	United States	10029
11	James Cancer Hospital and Solove Research Institute - 460 W 10th Ave	Columbus	Ohio	United States	43210-1240
12	Oncology Associates of Oregon, P.C.	Eugene	Oregon	United States	97401-6043
13	West Penn Hospital	Pittsburgh	Pennsylvania	United States	15224
14	Texas Oncology - Amarillo	Amarillo	Texas	United States	79106-1781
15	North Houston Cancer Clinics - Huntsville	Huntsville	Texas	United States	77340-4101
16	Wheeling Hospital Schiffler Cancer Center	Wheeling	West Virginia	United States	26003-6379
17	Froedtert and The Medical College of Wisconsin	Milwaukee	Wisconsin	United States	53226
18	Local Institution - 0016	Leuven	Vlaams Brabant	Belgium	3000
19	Local Institution - 0008	Roeselare	West-Vlaanderen	Belgium	8800
20	Local Institution - 0004	Praha 2	Praha, Hlavní Mesto	Czechia	128 21
21	Local Institution - 0023	Praha	Praha, Hlavní Mesto	Czechia	128 20
22	Local Institution - 0041	Nice	Alpes-Maritimes	France	06202
23	Local Institution - 0044	Tours	Indre-et-Loire	France	37044
24	Local Institution - 0026	Angers		France	49933
25	Local Institution - 0007	Grenoble cedex 09		France	38 38043
26	Local Institution - 0046	Paris		France	75475
27	Local Institution - 0053	Pierre Benite		France	69495
28	Local Institution - 0001	Poitiers		France	86021
29	Local Institution - 0013	München	Bayern	Germany	81675
30	Local Institution - 0040	Hannover	Niedersachsen	Germany	30161
31	Local Institution - 0037	Gütersloh	Nordrhein-Westfalen	Germany	33332
32	Local Institution - 0009	Leipzig	Sachsen	Germany	04103
33	Local Institution - 0021	Reggio Calabria	Calabria	Italy	89124
34	Local Institution - 0050	Roma	Lazio	Italy	00133
35	Local Institution - 0014	Novara	Piemonte	Italy	28100
36	Local Institution - 0045	Torino	Piemonte	Italy	10128
37	Local Institution - 0024	Firenze	Toscana	Italy	50139
38	Local Institution - 0015	Pavia		Italy	27100
39	Local Institution - 0029	Rozzano (MI)		Italy	20089
40	Local Institution - 0002	Wroclaw	Dolnoslaskie	Poland	50-556
41	Local Institution - 0030	Lublin	Lubelskie	Poland	20-081
42	Local Institution - 0049	Lodz	Lódzkie	Poland	93-513
43	Local Institution - 0010	Warszawa	Mazowieckie	Poland	02-172
44	Local Institution - 0034	Katowice	Slaskie	Poland	40-519
45	Local Institution - 0035	Wałbrzych		Poland	58-309
46	Auxilio Mutuo Cancer Center	San Juan		Puerto Rico	00917-5032
47	Local Institution - 0017	L'Hospitalet de Llobregat	Barcelona	Spain	08908
48	Local Institution - 0005	Barcelona		Spain	08035
49	Local Institution - 0052	Barcelona		Spain	08916
50	Local Institution - 0039	Granada		Spain	18014
51	Local Institution - 0038	Madrid		Spain	28006
52	Local Institution - 0027	Ourense		Spain	32005
53	Local Institution - 0028	Salamanca		Spain	37007
54	Local Institution - 0018	Valencia		Spain	46010