The goal of this clinical trial is to study the efficacy of the tagraxofusp + venetoclax combination in treatment-naive blastic plasmacytoid dendritic cell neoplasm adult patients.
The main question is to verify the response in patients after 3 cycles of tagraxofusp+venetolax and to demonstrate if the combination of tagraxofusp + venetoclax increases the rate of complete remission, assessed after 3 months of treatment.
Patients will receive a ramp-up phase of venetoclax during 3 days and at least 3 cycles of venetoclax. After, the investigators will evaluate the response, and depending on the response observed, patients may receive additional cycles of treatment for a maximum of 24 cycles, or receive an allograft or discontinue the treatment in the case of therapeutic failure.
Patients with a confirmed BPDCN diagnosis according to WHO 2022 revised criteria and have not received previous treatment : patients with skin or lymph node lesions but no bone marrow involvement can be included
Age >18 years
Ability to understand the protocol and to sign an informed consent
Possibility of follow-up
Participation to another clinical trial with any investigative drug within 30 days prior to study enrolment.
Previous treatment with venetoclax or tagraxofusp
Treatment of BPDCN with any prior chemotherapy or investigational agents, except hydroxyurea for less than 14 days at the time of inclusion
Concomitant immunosuppressive therapy -except for low-dose prednisone (≤10 mg/day)
Single group
Venetoclax
DrugSingle interventional arm
Tagraxofusp
DrugSingle interventional arm
Proportion of participants who achieve a cCR (CR or Cri or CRc) after 3 TAGVEN cycles
Evaluation will be done with a bone marrow aspirate, cytogenetics, metabolic imaging, study of MRD by NGS and flow-cytometry and SWAT SCORE
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French Innovative Leukemia Organisation
Lead sponsor
Stemline Therapeutics, Inc.
Collaborator
AbbVie
Collaborator
