Clinical trials with new drugs for β-thalassemias
| Drug trial name . | Trial no. . | Phase . | Purpose . | Disease . | No. of patients . | Status . |
|---|---|---|---|---|---|---|
| Luspatercept | NCT01749540/Extension NCT02268409 | 2 | Open-label, ascending dose study to evaluate the effects | TDT and NTDT | 64 (30 TDT, 34 NTDT) | Completed/active, not recruiting |
| Luspatercept BELIEVE Study | NCT02604433 | 3 | Double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety plus BSC vs placebo plus BSC | TDT | 300 | Active, not recruiting |
| Sotatercept (ACE-011) | NCT01571635 | 2A | Dose-finding study to determine safety and tolerability | TDT and NTDT | 46 | Active, not recruiting |
| Ruxolitinib TRUTH Study | NCT02049450 | 2 | Study of efficacy and safety | TDT | 30 | Completed |
| Drug trial name . | Trial no. . | Phase . | Purpose . | Disease . | No. of patients . | Status . |
|---|---|---|---|---|---|---|
| Luspatercept | NCT01749540/Extension NCT02268409 | 2 | Open-label, ascending dose study to evaluate the effects | TDT and NTDT | 64 (30 TDT, 34 NTDT) | Completed/active, not recruiting |
| Luspatercept BELIEVE Study | NCT02604433 | 3 | Double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety plus BSC vs placebo plus BSC | TDT | 300 | Active, not recruiting |
| Sotatercept (ACE-011) | NCT01571635 | 2A | Dose-finding study to determine safety and tolerability | TDT and NTDT | 46 | Active, not recruiting |
| Ruxolitinib TRUTH Study | NCT02049450 | 2 | Study of efficacy and safety | TDT | 30 | Completed |
BSC, best supportive care.