Table 2 Novel Therapeutics in β-Thalassemia
From: Erythropoiesis: insights into pathophysiology and treatments in 2017
| Company | Drug/Target | Mechanism | Eligibility | Route | Clintrials.gov | Status |
|---|---|---|---|---|---|---|
| a. Gene therapy | ||||||
| Bluebird Bio | BB305 lentiviral vector (betibeglogene darolentivec) | Improved erythropoiesis |
- Transfusion-dependent β-Thalassemia -age 12–35 | IV | NCT01745120 |
Active, not recruiting - Phase 1/2 |
| IRCCS San Raffaele | GLOBE lentiviral vector | Improved erythropoiesis |
- Transfusion-dependent β-Thalassemia -age ≥ 3 and < 65 | IV | NCT02453477 |
Open - Phase 1/2 |
| b. Small molecule targets | ||||||
| New England Research Institutes | Decitabine | HbF induction |
- age ≥ 18 - TD β Thalassemia and HbEβ-Thalassemia | Subcutaneously | NCT00661726 |
Completed - Phase 2 |
| Medical College Kolkata | Decitabine | HbF induction |
- age ≥ 18 - TDT and NDTD HbEβ-Thalassemia | Subcutaneously | – |
Completed - ASH 2017 |
| Novartis Pharmaceuticals | INC424 (Ruxolitinib) | Jak 1/2 inhibitor |
- age ≥ 18 - TDT β- Thalassemia - Splenomegaly - iron chelation × 4 weeks | Oral | NCT02049450 |
Completed - Phase 2a |
| Acceleron | ACE-536 (Luspatercept) | Ligand trap TBG beta superfamily |
- age ≥ 18 - TD and NTDT β-Thalassemia | Subcutaneously | NCT02268409 |
Active, not recruiting - Phase 2 |
| Celgene | ACE-011 (Sotatercept) | Ligand trap TBG beta superfamily |
- age ≥ 18 - TD and NTDT β-Thalassemia | Subcutaneously | NCT01571635 |
Active, not recruiting - Phase 2a |