Ongoing Clinical Trials for Blastic Plasmacytoid Dendritic Cell Neoplasm
There are currently 2 ongoing clinical trials investigating new treatment approaches for blastic plasmacytoid dendritic cell neoplasm, a rare and aggressive type of blood cancer. These trials are being conducted in France and the Netherlands, testing innovative drug combinations and targeted therapies aimed at improving outcomes for patients with this challenging condition.
Clinical trial locations
- France
- Netherlands
Study on the Effectiveness and Safety of Tagraxofusp and Venetoclax for Adults with Untreated Blastic Plasmacytoid Dendritic Cell Neoplasm
This trial is testing a combination of two medications, tagraxofusp and venetoclax, specifically for patients who have not yet received any treatment for their condition. The study is being conducted in France.
Main inclusion criteria: To participate, patients must be adults over 18 years old with a confirmed diagnosis according to the World Health Organization 2022 criteria. They must not have received any previous treatment for this condition. Patients need to have adequate kidney function with a creatinine clearance of at least 45 mL/min, adequate heart function with a left ventricular ejection fraction of 50% or higher, and an albumin level of at least 3.2 g/dL. They must also have adequate liver function and an Eastern Cooperative Oncology Group performance status of less than 3. Men and women who can have children must use highly effective birth control, and women must have a negative pregnancy test before starting treatment.
Main exclusion criteria: Patients cannot participate if they have other serious medical conditions that could interfere with the study, are pregnant or breastfeeding, or have had another type of cancer within the last 2 years (except certain skin cancers). Patients with active infections requiring treatment, those who have had major surgery within the last 4 weeks, or those with known allergies to the study drugs are also excluded. Additionally, patients currently participating in another clinical trial, those with a history of drug or alcohol abuse within the last 6 months, or those with mental health conditions that could interfere with participation are not eligible.
Focus and goal: The primary goal of this trial is to evaluate how well tagraxofusp and venetoclax work together in patients who have not received any previous treatment. The study aims to determine the rate of complete remission, meaning the cancer is no longer detectable, or a significant reduction in cancer symptoms after three cycles of treatment. Researchers will also monitor how long patients remain free from cancer progression and assess overall survival rates.
Investigational drugs: Tagraxofusp is administered as an intravenous infusion and works by targeting and attaching to a specific protein found on the surface of cancer cells, helping to destroy them. Venetoclax is taken orally as a tablet and works by blocking a protein that prevents cancer cells from dying, encouraging the cancer cells to die naturally. This combination approach is designed to enhance the overall effectiveness of the treatment.
Study of SAR443579 Infusion for Adults and Children with Relapsed or Refractory Acute Myeloid Leukemia, B-Cell Acute Lymphoblastic Leukemia, HR-MDS, or BPDCN
This trial is investigating a new treatment called SAR443579 for patients with several types of blood cancers, including blastic plasmacytoid dendritic cell neoplasm. The study is being conducted in France and the Netherlands and includes both adults and children.
Main inclusion criteria: For this condition specifically, children aged 1 to 17 years and adults in the escalation part of the study must have a confirmed diagnosis with relapsed or refractory disease and no available effective therapy. Participants must weigh at least 10 kg. All participants must be at least 1 year old and able to follow study procedures. The trial also includes patients with other types of blood cancers such as acute myeloid leukemia, B-cell acute lymphoblastic leukemia, and high-risk myelodysplastic syndrome, with specific criteria for each condition.
Main exclusion criteria: Patients with other types of cancer not part of the study are excluded, as well as those who have had different cancer treatments recently. Patients with uncontrolled serious infections, significant heart problems, or severe liver or kidney issues cannot participate. Pregnant or breastfeeding women are not eligible, nor are patients unable to follow study procedures. Those with a history of allergic reactions to similar treatments or who have participated in another clinical trial recently are also excluded.
Focus and goal: The study is divided into two parts. The first part focuses on determining the best and safest dose of SAR443579 for both adults and children by identifying the maximum tolerated dose. The second part aims to assess how well the treatment works at the recommended dose by evaluating its anti-leukemic activity. Throughout the study, participants will be closely monitored for safety and effectiveness.
Investigational drug: SAR443579 is administered as an intravenous infusion and works by targeting specific pathways involved in the growth and survival of cancer cells, aiming to inhibit their proliferation. It is currently classified as an investigational anti-cancer agent, and its effectiveness and safety are still being evaluated through this clinical research.
Summary
The two ongoing clinical trials for blastic plasmacytoid dendritic cell neoplasm reflect different treatment approaches for this rare and aggressive blood cancer. One trial focuses on previously untreated patients using a combination therapy approach, while the other targets patients whose disease has relapsed or not responded to treatment. France is involved in both trials, while the Netherlands participates in one. The trials test different mechanisms of action: tagraxofusp and venetoclax work through targeted cell destruction and promoting cancer cell death, while SAR443579 targets specific cancer cell growth pathways. Together, these studies represent important efforts to find more effective treatments for this challenging condition, with options available for both newly diagnosed and relapsed patients, as well as inclusion of pediatric populations in one of the trials.
