Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ACE-536 · 2 trials · 6 indications
Percentage of participants who become RBC-transfusion free over any consecutive 12-week period starting in first 24 weeks.
Determine the recommended dose of luspatercept that is safe and tolerable in pediatric participants with transfusion-dependent B-thalassemia or non-transfusion-dependent β-thalassemia
Maximum serum concentration of drug
Area under the curve
Half-life
Apparent oral clearance
Apparent volume of distribution
| Arm | Type | Description |
|---|---|---|
| Experimental Arm: Luspatercept (ACE-536) | EXPERIMENTAL | Luspatercept will be given to participants via subcutaneous injection (administered on Day 1 of each 21-day treatment cycle) |
| Control Arm: Placebo | PLACEBO_COMPARATOR | Placebo starting dose with volume equivalent to experimental arm subcutaneous injection every 3 weeks (administered on Day 1 of each 21-day treatment cycle) |
| Cohort 1: TD Dose Escalation Cohort: 12 to < 18 years Luspatercept 0.75 mg/kg | EXPERIMENTAL | - |
| Cohort 2: TD Dose Escalation Cohort: 12 to < 18 years: Luspatercept 1.0 mg/kg | EXPERIMENTAL | - |
| Cohort 3: TD Dose Expansion Cohort: 12 to <18 years Luspatercept 1.0 mg/kg | EXPERIMENTAL | - |
| Cohort 4: TD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.0 mg/kg | EXPERIMENTAL | - |
| Cohort 5: TD Dose Escalation Cohort: 6 to <12 years Luspatercept 1.2 mg/kg | EXPERIMENTAL | - |
| Cohort 6: NTD Dose Confirmation Cohort: 12 to < 18 years Luspatercept 1.0 mg/kg | EXPERIMENTAL | - |
| Cohort 7: NTD Dose Expansion Cohort: NTD 12 to < 18 years | EXPERIMENTAL | - |
| Cohort 8: NTD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.0 mg/kg | EXPERIMENTAL | - |
| Cohort 9: NTD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.2 mg/kg | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| ACE-536 | DRUG | Subcutaneous Injection |
| Placebo | OTHER | Subcutaneous Injection |
Subjects must satisfy the following criteria to be randomized in the study: Inclusion Criteria \- Subject is ≥18 years of age at the time of signing the ICF. * Subject has a diagnosis of PMF according to the 2016 World Health Organization (WHO) criteria or diagnosis of post-ET or post-PV MF accor...