Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

RecruitingRare disease

Study on the Effectiveness and Safety of Tagraxofusp and Venetoclax for Adults with Untreated Blastic Plasmacytoid Dendritic Cell Neoplasm

Fast replyRegistered drug
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare type of blood cancer called Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). The study will evaluate the effectiveness and safety of a combination of two treatments: tagraxofusp and venetoclax. Tagraxofusp is a medication that targets specific cells in the body, while venetoclax is a type of drug known as a BCL-2 inhibitor, which helps to kill cancer cells. The purpose of the study is to see how well these two medications work together in patients who have not received any previous treatment for BPDCN.

Participants in the study will receive both tagraxofusp and venetoclax over a period of time. Tagraxofusp is given as an infusion, which means it is delivered directly into the bloodstream through a vein, while venetoclax is taken orally as a tablet. The study will monitor how patients respond to the treatment combination, looking for signs of improvement in their condition. The trial will also assess the safety of the treatment, checking for any side effects that may occur.

The study aims to determine the rate of complete remission, which means the cancer is no longer detectable, or a significant reduction in cancer symptoms after three cycles of treatment. Researchers will also look at other outcomes, such as how long patients remain free from cancer progression and overall survival rates. This trial is an important step in finding more effective treatments for BPDCN and improving the quality of life for those affected by this rare cancer.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Beginning of the trial

    Upon joining the clinical trial, the patient will start the treatment with a combination of two medications: tagraxofusp and venetoclax. These medications are used to treat a condition known as blastic plasmacytoid dendritic cell neoplasm (BPDCN).

  2. Step 2

    Medication administration

    Tagraxofusp is administered through an intravenous (IV) infusion. This means the medication is given directly into the bloodstream through a vein. The specific dosage and frequency of administration will be determined by the healthcare team based on the patient's individual needs.

    Venetoclax is taken orally in the form of film-coated tablets. The dosage and frequency will also be determined by the healthcare team. It is important to follow the instructions provided by the healthcare team regarding how and when to take this medication.

  3. Step 3

    Treatment cycles

    The treatment is organized into cycles. Each cycle consists of a specific period during which the medications are administered, followed by a rest period. The primary goal is to evaluate the effectiveness and safety of the medication combination after three cycles.

    During these cycles, the healthcare team will monitor the patient's response to the treatment and any side effects that may occur. This monitoring is crucial to ensure the patient's safety and to make any necessary adjustments to the treatment plan.

  4. Step 4

    Evaluation of response

    After completing three cycles of treatment, the healthcare team will assess the patient's response. The main objective is to determine if there is a complete remission, which means the absence of disease symptoms, or a significant reduction in skin lesions.

    The evaluation will include various tests and assessments to measure the effectiveness of the treatment and to check for any remaining disease activity.

  5. Step 5

    Ongoing monitoring

    Throughout the trial, the patient will undergo regular check-ups and tests to monitor their health and the progress of the treatment. This includes blood tests, imaging studies, and other assessments as needed.

    The healthcare team will provide guidance and support to manage any side effects and to ensure the patient's well-being during the trial.

Who can join the trial?

11 criteria

  • Patients must have a confirmed diagnosis of Blastic Plasmacytoid Dendritic Cell Neoplasms (BPDCN) according to the World Health Organization (WHO) 2022 criteria and have not received previous treatment.
  • Patients must be older than 18 years.
  • Patients must be able to understand the study details and sign an informed consent form.
  • Patients must be able to attend follow-up appointments.
  • Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status of less than 3. This is a scale used to assess how a patient's disease is progressing and how the disease affects their daily living abilities.
  • Patients must have adequate kidney function, shown by a creatinine clearance of at least 45 mL/min. This is a measure of how well the kidneys are working.
  • Patients must have adequate heart function, defined by a Left Ventricular Ejection Fraction (LVEF) of 50% or higher, as measured by a heart scan (MUGA or ECHO), and no significant abnormalities on an electrocardiogram (ECG).
  • Patients must have an albumin level of at least 3.2 g/dL. Albumin is a protein in the blood that helps maintain fluid balance.
  • Patients must have adequate liver function.
  • Men and women who can have children must use a highly effective method of birth control.
  • Women must have a negative urine or blood pregnancy test within one week before starting treatment.

Who cannot join the trial?

10 criteria

  • Patients who have any other serious medical conditions that could interfere with the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who have had another type of cancer within the last 2 years, except for certain skin cancers.
  • Patients who have an active infection that requires treatment.
  • Patients who have had a major surgery within the last 4 weeks.
  • Patients who have a known allergy to the study drugs or similar drugs.
  • Patients who are currently participating in another clinical trial.
  • Patients who have a history of drug or alcohol abuse within the last 6 months.
  • Patients who have a mental health condition that could interfere with their ability to participate in the study.
  • Patients who have received certain treatments for their condition within a specific time frame before the study.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

  • Tagraxofusp

    is a medication used in this clinical trial to treat a rare type of blood cancer called blastic plasmacytoid dendritic cell neoplasm (BPDCN). It works by targeting and killing cancer cells. Tagraxofusp is designed to attach to a specific protein found on the surface of these cancer cells, helping to destroy them and reduce the number of cancer cells in the body.

  • Venetoclax

    is another medication used in the trial, which helps to treat BPDCN by targeting a protein that prevents cancer cells from dying. By blocking this protein, venetoclax encourages the cancer cells to die naturally, which can help to reduce the number of cancer cells and slow down the progression of the disease. This medication is used in combination with tagraxofusp to enhance the overall effectiveness of the treatment.

What is already known about the treatment

  • Tagraxofusp

    Tagraxofusp is administered as an intravenous infusion, meaning it is given directly into the bloodstream through a vein. It is currently being studied in clinical trials for its effectiveness in treating blastic plasmacytoid dendritic cell neoplasms (BPDCN), a rare type of blood cancer. The medication works by targeting a specific protein on cancer cells, delivering a toxin that helps kill these cells. It is classified as a targeted therapy, which means it specifically attacks cancer cells while minimizing damage to normal cells.

  • Venetoclax

    Venetoclax is taken orally in the form of a tablet. It is being evaluated in combination with tagraxofusp for its potential to treat blastic plasmacytoid dendritic cell neoplasms (BPDCN). Venetoclax works by inhibiting a protein that helps cancer cells survive, leading to their death. It is classified as a BCL-2 inhibitor, which is a type of targeted therapy that focuses on disrupting the survival mechanisms of cancer cells.

Investigated diseases

Blastic Plasmacytoid Dendritic Cell Neoplasms – This is a rare type of blood cancer that originates from a specific type of immune cell known as plasmacytoid dendritic cells. It typically presents with skin lesions, which may appear as purple or red patches, nodules, or ulcers. The disease can progress to involve the bone marrow and blood, leading to symptoms such as fatigue, fever, and weight loss. As it advances, it may cause a decrease in blood cell counts, resulting in anemia, increased risk of infections, and bleeding issues. The progression of the disease can vary, with some cases remaining localized to the skin for a period, while others rapidly spread to other parts of the body. Understanding the behavior of this disease is crucial for managing its impact on the patient's health.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-511003-42-00Protocol codeTAGVENEstimated enrolment33 patientsSponsorFrench Innovative Leukemia Organization (F.I.L.O.).

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

ClinicalTrials.eu

Every clinical trial in Europe, in plain language.

Explore the full ecosystem of clinical research in Europe.

Search trials

Legal notice · Published by CTIN POLAND sp. z o.o., ul. rtm. Witolda Pileckiego 67/109, 02-781 Warsaw, Poland · KRS 0001111334 · REGON 528919042 · NIP 9512598637

© 2026 Clinical Trials EU – European Clinical Trials Information Network

GDPR compliance, ISO 9001 and ISO 27001 certified (LL-C Certification)

On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).