Table 1.
Clinical trials of novel treatment in β-thalassemia.
| Treatment Modality | Mechanism | Route | Phase | ClinicalTrials.gov (8 May 2021) | Status | Institution/ Developer |
|---|---|---|---|---|---|---|
| Luspatercept | Ligand trap TGF beta superfamily |
Subcutaneous | 2 | NCT01749540 | Completed | Acceleron Pharma, Celgene Corporation |
| 2, extension study 2 2 |
NCT02268409 NCT03342404 NCT04143724 |
Completed Open Not yet recruiting |
||||
| 3 | NCT02604433 | Completed | ||||
| Mitapivat | Pyruvate kinase activation | Oral | 2 | NCT03692052 | Open | Agios Pharmaceuticals |
| TMPRSS6-LRx | Matriptase-2 inhibition, hepcidin activation | Subcutaneous | 2 | NCT04059406 | Open | Ionis Pharmaceuticals |
| SLN124 | Matriptase-2 inhibition, hepcidin activation | Subcutaneous | 2 | NCT04718844 | Open | Silence Therapeutics plc |
| PTG-300 | Hepcidin analog | Subcutaneous | 2 | NCT04054921 | Completed | Protagonist Therapeutics, Inc. |
| VIT-2763 | Ferroportin inhibition | Oral | 2 | NCT04364269 | Open | Vifor (International) Inc. |
| IMR-687 | Phosphodiesterase 9 inhibition, HbF stimulation | Oral | 2 | NCT04411082 | Completed | Imara, Inc. |
| HSCT | Reduced intensity conditioning | Matched sibling donor | 1, 2 | NCT00920972, NCT01050855, NCT02435901 | Open Open Completed |
US, Canada US, Canada US, Canada |
| Family related matched donor or cord | 2 | NCT00408447 | Active not recruiting | Columbia University, USA | ||
| HSCT | Matched unrelated | 2 | NCT01049854 | Completed | New York Medical College, USA | |
| HSCT | Nonmyeloablative haploidentical | Haploidentical transplants | 1,2 | NCT00977691 | Active not recruiting | National Heart, Lung, and Blood Institute (NHLBI) |
| HSCT | Nonmyeloablative peripheral blood mobilized | Allogeneic peripheral blood stem cell | 1,2 | NCT02105766 | Open | National Heart, Lung, and Blood Institute (NHLBI) |
| HSCT | Umbilical cord blood | Umbilical cord stem cells | 1 | NCT02126046 | Open | Nanfang Hospital of Southern Medical University |
| HSCT CordIn™ | Umbilical cord blood-derived ex vivo stem cells | Ex vivo umbilical cord stem cells | 1 | NCT02504619 | Completed | Gamida Cell ltd |
| LentiGlobin BB305 vector | β-globin gene addition | Ex vivo autologous CD34+ stem cell transduction | 1 | NCT02151526 | Completed | bluebird bio, France |
| 1/2 3 |
NCT01745120 NCT03207009 |
Completed Open |
bluebird bio, Northstar Study |
|||
| TNS9.3.55 Lentiviral Vector | β-globin gene addition | Ex vivo autologous CD34+ stem cell transduction | 1 | NCT01639690 | Active not recruiting | Memorial Sloan Kettering Cancer Center, USA |
| GLOBE Lentiviral Vector | β-globin gene addition | Ex vivo autologous CD34+ stem cell transduction | 1 | NCT02453477 | Active not recruiting | IRCCS San Raffaele, Italy |
| CTX001 | BCL11A gene editing | Ex vivo autologous CD34+ stem cell transduction | 1/2 | NCT03655678 | Open | Vertex Pharmaceuticals Incorporated CRISPR Therapeutics |
| ST-400 | BCL11A gene editing | Ex vivo autologous CD34+ stem cell transduction | 1/2 | NCT03432364 | Active not recruiting | Sangamo Therapeutics Sanofi |