Clinical Trials in India / NCT05891249
Active, not recruiting
Phase 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
NCT05891249 · tracked via the Priya Life Science India tracker
Condition(s) studied
Anemia
Investigational drug(s) / intervention(s)
Luspatercept: Specified dose on specified days
Study 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
Eligibility
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 is excluded unless the treatment stopped at least 28 days prior to enrollment. Anticoagulant therapies used for prophylaxis for surgery or high-risk procedures as well as low-molecular-weight (LMW) heparin for superficial venous thrombosis and chronic aspirin are allowed.
* Cytotoxic agents or immunosuppressants or immunomodulatory drugs (IMiDs) ≤ 28 days prior to enrollment (ie, antithymocite globulin or cyclosporine or thalidomide).
MDS-RS Cohort
* MDS associated with del 5q cytogenetic abnormality.
* Secondary MDS, that is, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases.
* Participant has known clinically significant anemia due to iron, vitamin B12, or folate deficiencies; autoimmune or hereditary hemolytic anemia; or gastrointestinal bleeding.
* Iron deficiency to be determined by serum ferritin ≤ 15 micrograms per liter (μg/L) and additional testing if clinically indicated (for example, calculated transferrin saturation \[iron/total iron binding capacity ≤ 20%\] or bone marrow aspirate \[BMA\] stain for iron).
Primary outcome measure(s)
- β-Thal Cohort: Number of participants with treatment-related adverse events (AEs) of grade 3 or higher — Up to 57 weeks
- MDS-Ring Sideroblasts (RS) Cohort: Number of participants with treatment-related AEs of grade 3 or higher — Up to 54 weeks
Trial sites (9)
| Facility | City | Region | Status |
|---|---|---|---|
| Local Institution - 0002 | Ahmedabad | Gujarat | |
| Local Institution - 0001 | New Delhi | National Capital Territory of Delhi | |
| Local Institution - 0007 | Kolkata | West Bengal | |
| Local Institution - 0005 | Assam | India | |
| Local Institution - 0003 | Bangalore | India | |
| Local Institution - 0004 | Chandigarh | India | |
| Local Institution - 0010 | Delhi | India | |
| Local Institution - 0006 | Hyderabad | India | |
| Local Institution - 0008 | Mumbai | India |
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05891249 on ClinicalTrials.gov ↗ ← All trials in India