Table 2.

Selection of active clinical studies for BPDCN*

Clinical studyMechanism of actionPhase of studyNCT #Clinical status
BPDCN International Registry N/A N/A NCT05430971 Recruiting 
Study of venetoclax, a BCL2 antagonist, for patients with BPDCN BCL2 inhibition NCT03485547 Active, not recruiting 
Combination chemotherapy in patients with newly diagnosed BPDCN Multi-agent cytotoxic chemotherapy: idarubicin, methotrexate
L-asparaginase
Dexamethasone 
NCT03599960 Active, not recruiting 
Study of IMGN632 in patients with untreated BPDCN and relapsed/refractory BPDCN CD123 targeted NCT03386513 Active, not recruiting 
Venetclax, SL401, and chemotherapy for treatment of BPDCN BCL2
CD123
Cytotoxic chemotherapy 
NCT04216524 Recruiting 
Tagraxofusp in patients with CD123+ or with BPDCN-IPh-like AML CD123 targeted NCT04342962 Recruiting 
Safety and efficacy of CD123-targeted CAR-NK for relapsed/refractory AML or BPDCN CD123 targeted 1/2 NCT06006403 Recruiting 
First-in-human study of SAR443579 infusion R/R AML, B-cell ALL, high-risk MDS, or BPDCN CD123 targeted 1/2 NCT05086315 Recruiting 
Genetically modified T-cell immunotherapy in treating patients with R/R AML and persistent/recurrent BPDCN CD123 targeted NCT02159495 Recruiting 
Venetoclax and decitabine in R/R AML or high-risk MDS BCL2– and hypomethylating NCT03404193 Active, not recruiting 
Flotetuzumab for R/R CD123+ hematologic malignancies CD123 x CD3 targeted NCT04681105 Active, not recruiting 
A study of MGD024 R/R hematologic malignancies CD123 x CD3-bispecific NCT05362773 Recruiting 
Cord blood transplant, cyclophosphamide, fludarabine, TBI in high-risk hematologic diseases Immune-modulating + cytotoxic chemotherapy NCT06013423 Recruiting 
Clinical studyMechanism of actionPhase of studyNCT #Clinical status
BPDCN International Registry N/A N/A NCT05430971 Recruiting 
Study of venetoclax, a BCL2 antagonist, for patients with BPDCN BCL2 inhibition NCT03485547 Active, not recruiting 
Combination chemotherapy in patients with newly diagnosed BPDCN Multi-agent cytotoxic chemotherapy: idarubicin, methotrexate
L-asparaginase
Dexamethasone 
NCT03599960 Active, not recruiting 
Study of IMGN632 in patients with untreated BPDCN and relapsed/refractory BPDCN CD123 targeted NCT03386513 Active, not recruiting 
Venetclax, SL401, and chemotherapy for treatment of BPDCN BCL2
CD123
Cytotoxic chemotherapy 
NCT04216524 Recruiting 
Tagraxofusp in patients with CD123+ or with BPDCN-IPh-like AML CD123 targeted NCT04342962 Recruiting 
Safety and efficacy of CD123-targeted CAR-NK for relapsed/refractory AML or BPDCN CD123 targeted 1/2 NCT06006403 Recruiting 
First-in-human study of SAR443579 infusion R/R AML, B-cell ALL, high-risk MDS, or BPDCN CD123 targeted 1/2 NCT05086315 Recruiting 
Genetically modified T-cell immunotherapy in treating patients with R/R AML and persistent/recurrent BPDCN CD123 targeted NCT02159495 Recruiting 
Venetoclax and decitabine in R/R AML or high-risk MDS BCL2– and hypomethylating NCT03404193 Active, not recruiting 
Flotetuzumab for R/R CD123+ hematologic malignancies CD123 x CD3 targeted NCT04681105 Active, not recruiting 
A study of MGD024 R/R hematologic malignancies CD123 x CD3-bispecific NCT05362773 Recruiting 
Cord blood transplant, cyclophosphamide, fludarabine, TBI in high-risk hematologic diseases Immune-modulating + cytotoxic chemotherapy NCT06013423 Recruiting 
*

This table was created according to listings on clinicaltrials.gov, accessed July 2024 with search terms “blastic plasmacytoid dendritic cell neoplasm” and “BPDCN” in “conditions/disease” and “other terms” filter options. This table represents a selection of those studies listed on the website as either “active,” “recruiting,” or “active, not recruiting” and is not meant to be an exhaustive list of all clinical trials available all over the world, but rather a selection to provide an idea of ongoing or recently completed clinical trials in this rare disease field.

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