A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants
- Registration Number
- NCT06045689
- Lead Sponsor
- Bristol-Myers Squibb
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 100
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Cohort 1: erythropoiesis-stimulating agents (ESA) naïve Luspatercept - Cohort 2: ESA relapsed or refractory Luspatercept -
- Primary Outcome Measures
Name Time Method 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
Name Time Method Time to AML progression Up to 4 years 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 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 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 with adverse events (AEs) Up to 2 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 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 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 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 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 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 ≥ 1.5 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 - 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 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 acute myeloid leukemia (AML) progression Up to 4 years Time from treatment start date to death due to any cause Up to 4 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
Trial Locations
- Locations (52)
Local Institution - 0051
🇺🇸Los Alamitos, California, United States
Local Institution - 0033
🇺🇸New Haven, Connecticut, United States
Local Institution - 0055
🇺🇸Saint Petersburg, Florida, United States
Local Institution - 0056
🇺🇸Wellington, Florida, United States
Local Institution - 0020
🇺🇸Kansas City, Kansas, United States
Local Institution - 0025
🇺🇸Paducah, Kentucky, United States
Local Institution - 0011
🇺🇸Detroit, Michigan, United States
Local Institution - 0059
🇺🇸Saint Louis, Missouri, United States
Local Institution - 0058
🇺🇸Morristown, New Jersey, United States
Local Institution - 0032
🇺🇸New York, New York, United States
Scroll for more (42 remaining)Local Institution - 0051🇺🇸Los Alamitos, California, United States