Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Luspatercept · 4 trials · 4 indications
An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a study drug, which did not necessarily have a causal relationship with the treatment. An AE could therefore have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study drug whether or not it was considered related to the study drug. The number of participants who experienced an AE is reported.
An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a study drug, which did not necessarily have a causal relationship with the treatment. An AE could therefore have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study drug whether or not it was considered related to the study drug. The number of participants who discontinued study treatment due to an AE is reported.
An erythroid response in NTD participants was defined as a mean hemoglobin increase of ≥1.5 g/dL from baseline for ≥14 days in the absence of blood transfusion. Baseline hemoglobin for NTD participants was the average of two or more measurements performed during the screening period. Hemoglobin measurements within 2 weeks following red blood cell (RBC) transfusion or 56 days after the last dose were excluded from analysis. NTD participants were participants who had received \<4 units of RBCs within 8 weeks prior to the first dose of study drug. The percentage of participants with a hemoglobin increase of ≥1.5 g/dL from baseline for ≥14 days is presented.
Transfusion burden for TD participants was defined as the ratio of RBC transfusion units (or milliliters) transfused during a 12-week interval divided by the duration of that interval compared to the ratio of RBC transfusion units 12 weeks prior to the start of treatment (baseline). The interval during the pretreatment period was defined as the 12 weeks prior to the first dose of study drug. An interval during the treatment plus follow-up period was defined as any 12-week interval after the first dose of study drug. TD participants were participants who had received ≥4 units of RBCs every 8 weeks (confirmed over 6 months prior to the first dose of study drug). The percentage of participants with a ≥20% reduction in RBC transfusion burden from baseline is presented.
The mHI-E for LTB participants was defined as a hemoglobin increase of ≥1.5 g/dL from baseline for ≥14 days or rolling 2 weeks (in the absence of red blood cell \[RBC\] transfusions). Hemoglobin measurements within 7 days following RBC transfusion were excluded from analysis. LTB participants were those who received \<4 units of RBCs within 8 weeks prior to baseline. Rolling 2 weeks was defined as any consecutive 2 weeks during the study. The percentage of LTB participants with mHI-E were reported.
The mHI-E for HTB participants was defined as a ≥4 units or ≥50% reduction in RBC transfusion burden during any rolling 8-week window compared to baseline. HTB participants were those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline. Rolling 8 weeks was defined as any consecutive 8 weeks during the study. The percentage of HTB participants with mHI-E were reported.
| Arm | Type | Description |
|---|---|---|
| Luspatercept Extension Population | EXPERIMENTAL | Participants receive luspatercept 0.6, 0.8, 1.0, or 1.25 mg/kg administered by subcutaneous injection on Day 1 of each 21-day cycle for up to 87 cycles (up to approximately 5 years). Participants will receive the highest tolerated dose level of luspatercept that they were assigned in the base study unless a dose modification was required. |
| Luspatercept 0.2 mg/kg | EXPERIMENTAL | Participants receive luspatercept 0.2 mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Luspatercept 0.4 mg/kg | EXPERIMENTAL | Participants receive luspatercept 0.4 mg/kg as an SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Luspatercept 0.6 mg/kg | EXPERIMENTAL | Participants receive luspatercept 0.6 mg/kg as an SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Luspatercept 0.8 mg/kg | EXPERIMENTAL | Participants receive luspatercept 0.8 mg/kg as an SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Luspatercept 1.0 mg/kg | EXPERIMENTAL | Participants receive luspatercept 1.0 mg/kg as an SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Luspatercept 1.25 mg/kg | EXPERIMENTAL | Participants receive luspatercept 1.25 mg/kg as an SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Expansion Cohort | EXPERIMENTAL | Participants receive an initial dose of luspatercept 0.8 mg/kg as an SC injection on Day 1 of Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated up to a maximum dose of 1.25 mg/kg based on the safety review team (SRT) recommendations. |
| Luspatercept 0.125mg/kg (Cohort 1) | EXPERIMENTAL | Participants receive luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Luspatercept 0.25mg/kg (Cohort 2) | EXPERIMENTAL | Participants receive luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Luspatercept 0.50mg/kg (Cohort 3) | EXPERIMENTAL | Participants receive luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Luspatercept 0.75mg/kg (Cohort 4) | EXPERIMENTAL | Participants receive luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Luspatercept 1.00mg/kg (Cohort 5) | EXPERIMENTAL | Participants receive luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Luspatercept 1.33mg/kg (Cohort 6) | EXPERIMENTAL | Participants receive luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Luspatercept 1.75mg/kg (Cohort 7) | EXPERIMENTAL | Participants receive luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days). |
| Name | Type | Description |
|---|---|---|
| luspatercept | DRUG | subcutaneous injection |
The main inclusion and exclusion criteria include but are not limited to the following: Inclusion Criteria: * Completion of the treatment period in the base study A536-04. * Females of child- bearing potential (defined as sexually mature women who have not undergone hysterectomy or bilateral oopho...
Luspatercept is an investigational small molecule being developed for hematology conditions including β-Thalassemia, Myelodysplastic Syndromes, and Anemia. It is currently in Phase 2 clinical development and is not approved by the FDA.
Luspatercept is being developed by Merck & Company, Inc., which trades under the ticker MRK. The company is conducting clinical trials to evaluate the drug for hematology indications.
Luspatercept is in Phase 2 clinical development. It is an investigational drug and has not received FDA approval. Clinical trials are ongoing or completed to evaluate its safety and efficacy.
Luspatercept has been studied in several Phase 2 trials including NCT01749514 for Anemia in Myelodysplastic Syndrome, NCT01749540 for Beta-thalassemia, and extension studies NCT02268383 and NCT02268409. All trials are completed.
Yes, Luspatercept is also known as ACE-536. Clinical trial NCT01749540 references Luspatercept (ACE-536) in its title, confirming the alternative name for the drug.