A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion… (NCT05891249) | Clinical Trial Compass
Active — Not RecruitingPhase 4
A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India
India85 participantsStarted 2023-06-05
Plain-language summary
The purpose of this study is to evaluate the safety and effectiveness of luspatercept in participants who require regular blood cell transfusions due to b-thalassemia and myelodysplastic syndromes in India
Who can participate
Age range18 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
β-Thalassemia Cohort
* Documented diagnosis of β-thalassemia or hemoglobin (Hb E/β-thalassemia). (β-thalassemia with mutation and/or multiplication of alpha \[α\] globin is allowed).
* Regularly transfused, defined as 6 RBC units to 20 RBC units in the 24 weeks prior to enrollment and no transfusion-free period for \> 35 days during that period.
MDS-RS Cohort
\- Participant has documented diagnosis of MDS according to World Health Organization (WHO) (2016)/French-American-British FAB classification that meets revised International Prognostic Scoring System (IPSS-R) classification of very low, low, or intermediate risk disease and the following criteria: i) RS ≥ 15% of erythroid precursors in bone marrow. If the SF3B1 mutation is present, RS ≥ 5% will be included.
ii) Less than 5% blasts in bone marrow and \< 1% peripheral blood blasts. iii) Peripheral blood white blood cell (WBC) count \< 13,000/ microliters (μL).
* If the participant was previously treated with erythropoiesis-stimulating agents (ESAs) or granulocyte colony-stimulating factor (G-CSF)/granulocyte-macrophage colony-stimulating factor (GM-CSF), both agents must have been discontinued ≥ 4 weeks prior to the date of enrollment.
Exclusion Criteria:
β-Thalassemia Cohort
* A diagnosis of Hb S/β-thalassemia or α-thalassemia (for exampe, Hemoglobin H).
* Deep vein thrombosis (DVT) or stroke requiring medical intervention ≤ 24 weeks prior to enrollment.
* Use of chronic anticoagulant therapy…
What they're measuring
1
β-Thal Cohort: Number of participants with treatment-related adverse events (AEs) of grade 3 or higher
Timeframe: Up to 57 weeks
2
MDS-Ring Sideroblasts (RS) Cohort: Number of participants with treatment-related AEs of grade 3 or higher