A Phase 3b Study of BMS-986346 in Lower-risk Myelodysplastic Syndrome Participants
- Conditions
- Myelodysplastic syndromeMedDRA version: 20.0Level: HLTClassification code: 10028536Term: Myelodysplastic syndromes Class: 10029104Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2023-504541-31-00
- Lead Sponsor
- Celgene Corp.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 100
Participants are eligible to be included in the study if all following criteria apply: A) Participant must be 18 years or older, have documented diagnosis of MDS, have an Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2. For Cohort 1 only: participant must have an endogenous serum erythropoietin (EPO) level of < 500 U/L and no prior ESA treatment. For Cohort 2 only: participant must be refractory or intolerant to prior ESA treatment as defined by any of the following: i) Refractory to prior ESA treatment: Documentation of nonresponse or response that was no longer maintained to prior ESA-containing regimen, either as a single agent or in combination (eg, with G-CSF). The ESA regimen must have been either: (1) Recombinant human erythropoietin = 40,000 IU/week for at least 8 doses or equivalent; or 1050 mg/kg/week for at least 8 doses or equivalent OR (2) Darbepoetin- darbepoetin alpha = 240 µg every week for at least 12-weeks or equivalent ii) Intolerant to prior ESA treatment: Documentation of discontinuation of prior ESA-containing regimen, either as a single agent or in combination (eg, with G-CSF), at any time after introduction due to intolerance or an AE. B) And must require RBC transfusion documented by the following criteria: i) Average transfusion requirement of = 1 units of packed RBCs (pRBCs) confirmed for a minimum of 8-weeks immediately prior to first treatment of luspatercept ii) Hb levels at the time of or within 7 days prior to administration of an RBC transfusion must be = 10.0 g/dL in order for the transfusion to be counted towards meeting eligibility criteria. RBC transfusions administered when Hb levels are > 10 g/dL and/or RBC transfusions administered for elective surgery do not qualify as a required transfusion for the purpose of meeting eligibility criteria
A participant will be excluded from the study if they have a secondary MDS, known history of diagnosis of AML, prior allogenic or autologous stem cell transplant, history of allergy or hypersensitivity to study drug components, pregnant, plan to get pregnant or breastfeeding, prior history of malignancies, known significant anemia due to iron, vitamin B12 or folate deficiencies.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective: to evaluate red blood cell transfusion independence (RBC-TI) with an associated concurrent mean hemoglobin (Hb) increase of = 1 g/dL after luspatercept initiated at the maximum approved dose for the treatment of anemia due to IPSS-R very low-, low, or intermediate- risk who require RBC transfusions.;Secondary Objective: Main secondary efficacy objective: evaluate the effect of luspatercept on reduction in RBC transfusions and increase in Hb, duration of RBC-TI, and time to RBC TI in MDS participants. Main secondary safety objective: to assess the safety and tolerability of luspatercept in MDS participants using a dosing regimen starting at the highest approved dose of 1.75 mg/kg.;Primary end point(s): Primary endpoint: composite of achievement of RBC-TI for 8-weeks with a concurrent mean Hb increase of = 1 g/dL from Week 1 to Week 24.
- Secondary Outcome Measures
Name Time Method Secondary end point(s):Main secondary efficacy endpoints: 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 from first dose to first onset of RBC TI = 8, 12, and 16 weeks, Maximum duration of RBC-TI for participants who achieve RBC TI = 8- and 16-week period;Secondary end point(s):Main secondary safety endpoint is the type, frequency, severity of AEs, and relationship of adverse events (AEs) to luspatercept from screening to 6 weeks (42-days) post last dose and from Week 1 to Week 48