Table 1.

Selected current clinical trials in novel therapeutics

NCT numberStudy titleConditionsInterventionsResults
NCT05885555 A Study of Ianalumab (VAY736) in Patients with Primary Immune Thrombocytopenia (ITP) Previously Treated with at Least Two Lines of Therapies Primary immune thrombocytopenia (ITP) Ianalumab Pending 
NCT05653349 Study of Ianalumab versus Placebo in Addition to First-Line Corticosteroids in Primary Immune Thrombocytopenia (ITP) Primary ITP Ianalumab vs placebo or corticosteroids Pending 
NCT05338190 Efficacy and Safety of Subcutaneous Belimumab or Placebo in Addition of Rituximab in Persistent or Chronic Immune Thrombocytopenia Primary ITP Rituximab with subcutaneous belimumab | Rituximab with subcutaneous placebo Pending 
NCT04562766 Study to Evaluate Rilzabrutinib in Adults and Adolescents with Persistent or Chronic Immune Thrombocytopenia (ITP) ITP Rilzabrutinib vs placebo Pending 
NCT05599880 The Investigate Efficacy and Safety Evaluation of Bortezomib in Patients with Relapsed/Refractory Immune Thrombocytopenia Relapsed/ refractory ITP Bortezomib Pending 
NCT06281327 Avatrombopag in the Treatment of Pediatric Immune Thrombocytopenia Persistent or chronic pediatric ITP Avatrombopab vs placebo Pending 
NCT03154385 Efficacy and Safety Assessment of a Treatment Combining Rituximab and Belimumab in Adults with Persistent Immune Thrombocytopenia (RITUX PLUS) Persistent and chronic ITP Belimumab with rituximab Primary: 80% CR + R at 52 weeks with 66.7% with CR; secondary: 86.7% initial overall response (13/15).
Safety: primarily related to rituximab infusions; 8 AE possibly related to tx but no SAE. 
NCT03275454 A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple-Dose BIVV009 in Participants With Chronic Immune Thrombocytopenia (ITP) Chronic ITP Sutimlimab Primary: safety: 1 serious not related to drug; 1 serious related to drug (migraine).
Efficacy: 42% R and CR in 33%; rapid response (~24 hrs) 
NCT03102593 A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients with ITP Chronic ITP Efgartigimod Primary: safety: 1 SAE worsening thrombocytopenia leading to discontinuation; extension: 2 SAE: pneumonia and endometrial polyposis.
Efficacy: CR 38.5% 5  mg/kg arm; 38.5% 10  mg/kg arm; 16.7% placebo 
NCT03395210 A Study of Rilzabrutinib in Adult Patients with Immune Thrombocytopenia (ITP) Chronic, refractory ITP Rilzabruitinib vs place Primary: 26.7% (CR or R) and durable; entered LTE at 400 mg BID and of those 92% continue to have plt ≥50 × 109/L.
Safety: 4 patients with respiratory infections (COVID-19, bronchitis, COVID-19 pneumonia); 3 serious AE: COVID-19 pneumonia; grade 4 thrombocytopenia; grade 3 pneumonia/pulmonary embolism 
NCT numberStudy titleConditionsInterventionsResults
NCT05885555 A Study of Ianalumab (VAY736) in Patients with Primary Immune Thrombocytopenia (ITP) Previously Treated with at Least Two Lines of Therapies Primary immune thrombocytopenia (ITP) Ianalumab Pending 
NCT05653349 Study of Ianalumab versus Placebo in Addition to First-Line Corticosteroids in Primary Immune Thrombocytopenia (ITP) Primary ITP Ianalumab vs placebo or corticosteroids Pending 
NCT05338190 Efficacy and Safety of Subcutaneous Belimumab or Placebo in Addition of Rituximab in Persistent or Chronic Immune Thrombocytopenia Primary ITP Rituximab with subcutaneous belimumab | Rituximab with subcutaneous placebo Pending 
NCT04562766 Study to Evaluate Rilzabrutinib in Adults and Adolescents with Persistent or Chronic Immune Thrombocytopenia (ITP) ITP Rilzabrutinib vs placebo Pending 
NCT05599880 The Investigate Efficacy and Safety Evaluation of Bortezomib in Patients with Relapsed/Refractory Immune Thrombocytopenia Relapsed/ refractory ITP Bortezomib Pending 
NCT06281327 Avatrombopag in the Treatment of Pediatric Immune Thrombocytopenia Persistent or chronic pediatric ITP Avatrombopab vs placebo Pending 
NCT03154385 Efficacy and Safety Assessment of a Treatment Combining Rituximab and Belimumab in Adults with Persistent Immune Thrombocytopenia (RITUX PLUS) Persistent and chronic ITP Belimumab with rituximab Primary: 80% CR + R at 52 weeks with 66.7% with CR; secondary: 86.7% initial overall response (13/15).
Safety: primarily related to rituximab infusions; 8 AE possibly related to tx but no SAE. 
NCT03275454 A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple-Dose BIVV009 in Participants With Chronic Immune Thrombocytopenia (ITP) Chronic ITP Sutimlimab Primary: safety: 1 serious not related to drug; 1 serious related to drug (migraine).
Efficacy: 42% R and CR in 33%; rapid response (~24 hrs) 
NCT03102593 A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients with ITP Chronic ITP Efgartigimod Primary: safety: 1 SAE worsening thrombocytopenia leading to discontinuation; extension: 2 SAE: pneumonia and endometrial polyposis.
Efficacy: CR 38.5% 5  mg/kg arm; 38.5% 10  mg/kg arm; 16.7% placebo 
NCT03395210 A Study of Rilzabrutinib in Adult Patients with Immune Thrombocytopenia (ITP) Chronic, refractory ITP Rilzabruitinib vs place Primary: 26.7% (CR or R) and durable; entered LTE at 400 mg BID and of those 92% continue to have plt ≥50 × 109/L.
Safety: 4 patients with respiratory infections (COVID-19, bronchitis, COVID-19 pneumonia); 3 serious AE: COVID-19 pneumonia; grade 4 thrombocytopenia; grade 3 pneumonia/pulmonary embolism 

AE, adverse event; CR, complete response; LTE, long-term efficacy; plt, platelet; R, response; SAE, severe adverse event; tx, treatment.

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