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.
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 outcome:
1. 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 (Time Frame - Up to week 24)
Secondary outcome:
1. 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 (Time Frame - Up to week 48)
2. 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 (Time Frame - Up to 2 years)
3. 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 (Time Frame - Up to 2 years)
4. 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 (Time Frame - Up to 2 years)
5. 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 (Time Frame - Up to 2 years)
6. 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 (Time Frame - Up to 2 years)
7. 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 (Time Frame - Up to 2 years)
8. 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 (Time Frame - Up to 2 years)
9. 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 (Time Frame - Up to 2 years)
10. 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 (Time Frame - Up to 2 years)
11. 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 (Time Frame - Up to 2 years)
12. 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 (Time Frame - Up to week 48)
13. 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 (Time Frame - Up to week 48)
14. Number of participants with adverse events (AEs) (Time Frame - Up to 2 years)
15. Number of participants with acute myeloid leukemia (AML) progression (Time Frame - Up to 4 years)
16. Time to AML progression (Time Frame - Up to 4 years)
17. Time from treatment start date to death due to any cause (Time Frame - Up to 4 years)
18. 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 (Time Frame - Up to 2 years)
- Experimental: Cohort 1: erythropoiesis-stimulating agents (ESA) naïve
- Experimental: Cohort 2: ESA relapsed or refractory
- Luspatercept (BMS-986346 / ACE-536 / REBLOZYL / ):
Specified dose on specified days.
Quelle: ClinicalTrials.gov
