Tagraxofusp and venetoclax in newly diagnosed BPDCN

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase 2 study evaluates the safety and efficacy of a tagraxofusp–venetoclax regimen in treatment-naive adults with Blastic Plasmacytoid Dendritic Cell Neoplasm. Participants receive a venetoclax ramp-up over 3 days, followed by at least 3 cycles of therapy, with the possibility of additional cycles, an allograft, or discontinuation for therapeutic failure based on response after 3 cycles and at 3 months. The primary goal is to determine the rate of complete remission after treatment and to assess overall response in relation to the combination therapy. Response assessments guide subsequent treatment decisions up to a maximum of 24 cycles. Safety and tolerability are monitored throughout the study.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. 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
2. Age \>18 years
3. Ability to understand the protocol and to sign an informed consent
4. Possibility of follow-up
5. ECOG \< 3
6. Adequate renal function as demonstrated by a calculated creatinine clearance ≥ 60 mL/min by the Cockcroft-Gault formula.
7. Adequate cardiac function defined by LVEF \>/= 50% by MUGA or ECHO and no clinically significant abnormalities on a 12-lead ECG
8. Albumin level≥3,2g/dL
9. Adequate liver function as demonstrated by:

   * aspartate aminotransferase (AST) ≤ 2.5 × ULN\*
   * alanine aminotransferase (ALT) ≤ 2.5 × ULN\*
   * bilirubin ≤ 3.0 × ULN, unless due to Gilbert's syndrome\* \* Unless considered due to leukemic organ involvement, in that cases values must be ≤ 10 × ULN
10. Men, and women of childbearing potential must be using a highly effective method of contraception
11. Negative urine/blood pregnancy test within 1 week prior to the initiation of treatment (if applicable)
12. Patient covered by any social security system

Exclusion Criteria:

1. Participation to another clinical trial with any investigative drug within 30 days prior to study enrolment.
2. Previous treatment with venetoclax or tagraxofusp
3. Treatment of BPDCN with any prior chemotherapy or investigational agents, except hydroxyurea for less than 14 days at the time of inclusion
4. Concomitant immunosuppressive therapy -except for low-dose prednisone (≤10 mg/day)
5. Known allergy or sensitivity to tagraxofusp, venetoclax, and any of its components or excipients.
6. Pregnant or breastfeeding woman
7. Known positivity for hepatitis B or C infection except for those subjects with an undetectable viral load or subjects with serologic evidence of prior vaccination to HBV
8. Evidence of uncontrolled systemic infection requiring therapy (viral, bacterial, or fungal)
9. Subject has any history of clinically significant condition(s) that in the opinion of the investigator would adversely affect his/her participating in this study including, but not limited to:

   * Cardiovascular disease e.g., NYHA heart failure \> class 2, uncontrolled angina, history of myocardial infarction, unstable angina, or stroke within 6 months prior to study entry, uncontrolled hypertension, or clinically significant arrythmias not controlled by medication.
   * Renal, pulmonary, neurologic, psychiatric, endocrinologic, metabolic, immunologic, hepatic, cardiovascular disease, or bleeding disorder independent of leukemia.
10. Subject with a history of other malignancies within the last three years prior to study entry, except for:

    * Adequately treated in situ carcinoma of the breast or cervix uteri
    * Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin
    * Prostate cancer without needs for specific therapy
    * Previous malignancy confined and surgically resected (or treated with other modalities) with curative intent.
11. Malabsorption syndrome or other conditions that preclude enteral route of administration
12. Patient with hereditary fructose intolerance

Where it's running

• AMIENS - CH Amiens Picardie Site Sud
  Amiens, France Recruiting
• ANGERS - CHU - Maladies du sang
  Angers, France Recruiting
• BESANCON - Hôpital Jean Minjoz - Hématologie
  Besançon, France Recruiting
• AP-HP- CRETEIL - CHU Henri Mondor
  Créteil, France Recruiting
• LILLE CHU - Hôpital Claude Huriez
  Lille, France Recruiting
• LYON HCL- Site Sud
  Lyon, France Recruiting

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• BPDCN patients treated with chemotherapy: a look back at real-world use

  This study tests how chemotherapy has performed in adults with BPDCN and compares the results to a prospective registry for ELZONRIS.

  Recruiting at 67 sites Stemline Therapeutics, Inc. · Aarhus and 66 other locations · NCT07785180

• Tagraxofusp in BPDCN: Real-world Registry of Safety and Effectiveness

  This registry follows adults with BPDCN to see how tagraxofusp works in everyday clinical practice and how safe it is over time.

  Recruiting at 41 sites Stemline Therapeutics, Inc. · Innsbruck and 40 other locations · NCT07785154

• International registry for Blastic Plasmacytoid Dendritic Cell Neoplasm

  This study is creating a global database for people with Blastic Plasmacytoid Dendritic Cell Neoplasm to gather information on their experiences and treatments to help improve care for this rare cancer.

  Recruiting at 22 sites Immune Oncology Research Institute · Miami, Florida and 21 other locations · NCT05430971

• CD123-targeted CAR-NK therapy for relapsed/refractory AML or BPDCN

  This study is testing a new treatment using special immune cells to see if it can help people with relapsed or hard-to-treat acute myeloid leukemia or blastic plasmacytoid dendritic cell neoplasm.

  Recruiting Phase 1 Chongqing Precision Biotech Co., Ltd · Wuhan, Hubei · NCT06690827

• Using cord blood transplant and chemotherapy to treat high-risk blood cancers

  This study is testing if combining umbilical cord blood transplants with certain chemotherapy treatments can help people with high-risk blood cancers do better and have fewer side effects.

  Recruiting Phase 2 Fred Hutchinson Cancer Center · Seattle, Washington · NCT06013423

• Treatment for Blastic Plasmacytoid Dendritic Cell Neoplasm using Venetoclax and SL-401

  This study is testing a combination of venetoclax, SL-401, and chemotherapy to see if it can help people with newly diagnosed blastic plasmacytoid dendritic cell neoplasm live longer and feel better.

  Recruiting Phase 2 M.D. Anderson Cancer Center · Houston, Texas · NCT04216524

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.