Post-Authorisation Registry of ELZONRIS (Tagraxofusp) in Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  ELZONRIS
• Study drug International non-proprietary name (INN) or common name:
  TAGRAXOFUSP
• Anatomical Therapeutic Chemical (ATC) code:
  (L01XX67) tagraxofusp

  tagraxofusp
• Medical condition to be studied:
  Blastic plasmacytoid dendritic cell neoplasia

Population studied

• Short description of the study population:
  Adult participants with BPDCN who are to be treated (or have recently started treatment) with tagraxofusp as first-line treatment according to the European Union summary of product characteristics in standard clinical practice setting across Europe. Participants who are relapse/refractory to prior treatment will also be investigated.
• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Estimated number of subjects:
  80

Study design details

• Study design:
  STML-401-0521 is a non-interventional study that primarily aims to provide clinical effectiveness and safety data of tagraxofusp when used in accordance with the summary of product characteristics in the post-marketing setting in participants with BPDCN.
• Main study objective:
  The main objective of this registry study is to investigate the effectiveness and safety of tagraxofusp in the treatment of adult participants with BPDCN in the real-world clinical practice setting. No study medication will be provided by the sponsor during this study.
• Setting:
  Key inclusion criteria for this study include:; • Diagnosed with BPDCN, confirmed by hematopathology and biomarkers (inclusive of CD123, CD4, and CD56).; • Planning to start treatment with tagraxofusp monotherapy for BPDCN at the time of, or within 6 months prior to, informed consent form (ICF) signature or has started treatment.; ; Key exclusion criteria for this study include:; • Currently (or planning to) participating in another study or registry where BPDCN treatment outcomes are reported.; • Participating in an interventional clinical trial at the time of ICF signature.; • Known allergic reactions to components of tagraxofusp.; ; Other protocol-specified criteria apply.
• Comparators:
  Not applicable
• Outcomes:
  The primary outcome measures include:; 1. Number of Participants Achieving Complete Response After 3 Months (+/- 1 month) of Treatment; 2. Number of Participants Experiencing Capillary Leak Syndrome; ; The secondary outcome measures include:; 1. Number of Participants Who Receive Stem Cell Transplant (SCT); 2. Progression-free Survival (PFS); 3. Overall Survival (OS); 4. Best Overall Response (BOR); 5. Duration of Response (DOR); 6. Number of Participants with Dose Interruptions and/or Requiring Administration of Intravenous Albumin Supplementation; 7. Number of Participants with Adverse Events of Special Interest (AESI); 8. Number of Participants with Adverse Events (AEs); 9. Number of Doses/Cycles of Tagraxofusp Administered