In short
There are currently 3 clinical trials underway in Poland investigating new approaches to diagnosing and treating histiocytosis, including Langerhans cell sarcoma. These studies focus on advanced imaging techniques and targeted medications for young patients whose condition has not responded to standard treatments.
Clinical trial locations
- Poland
Study on the Use of Fludeoxyglucose (18F) in PET/CT Scans for Young Patients with Histiocytosis
This trial explores the use of specialized imaging technology to better understand and monitor histiocytosis in young patients. The study focuses on using PET/CT scans with a radioactive sugar substance called Fludeoxyglucose (18F), which helps doctors visualize areas of high cell activity in the body.
Who can participate: This study is open to children and teenagers under 18 years old who have confirmed or suspected histiocytosis. The patient or their guardian must agree to participate by signing an informed consent form.
Who cannot participate: There are no specific exclusion criteria listed for this trial, making it broadly accessible to eligible young patients.
What the trial aims to achieve: The main goal is to determine whether PET/CT imaging with Fludeoxyglucose (18F) is both safe and beneficial for young patients with histiocytosis. Researchers will monitor how well the imaging technique works for tracking the disease and its response to treatment. The study will also evaluate event-free survival, progression-free survival, overall survival, and the rate at which the disease may return after two years.
Investigational drug: Fludeoxyglucose (18F-FDG) is a radioactive form of sugar used for imaging purposes. It is administered through an injection into a vein and helps highlight areas where cells are particularly active, which can indicate disease presence or progression.
Study on Trametinib Dimethyl Sulfoxide for Children with Refractory Histiocytosis Not Responding to Conventional Treatment
This clinical trial tests a medication called Trametinib for children whose histiocytosis has not improved with standard treatments. The study aims to find the right dose and treatment duration for young patients while evaluating the drug's safety and effectiveness.
Who can participate: Children and teenagers with histiocytosis who have not responded to previous treatments are eligible. Specifically, the disease must have worsened during or after treatment with medications like Vinblastine and prednisolone, or other second-line treatments such as Cytosine Arabinoside or Cladribine. Patients must either not have mutations in the BRAF gene or have previously tried Vemurafenib without success. The patient or guardian must sign an informed consent form, and participants must be enrolled in the HISTIOGEN trial. Both male and female patients can participate, and those of childbearing age must use effective contraception during treatment and for at least one year afterward.
Who cannot participate: Patients without histiocytic cell proliferation, those who are not children or teenagers, those who have not tried Vemurafenib (if BRAF positive), and those whose disease has responded to treatment cannot join this study.
What the trial aims to achieve: The study will assess how safe and effective Trametinib is for young patients with treatment-resistant histiocytosis. Researchers will monitor event-free survival, progression-free survival, overall survival, and overall response rates. The trial will also track the rate at which the disease reactivates after two years and document any adverse events. Regular health checks, laboratory tests, heart monitoring, and imaging will be conducted throughout the study.
Investigational drug: Trametinib is a targeted therapy taken as a tablet by mouth. It works by inhibiting a specific protein in the cell signaling pathway known as MEK, which helps slow or stop the growth of abnormal cells. The trial is exploring whether switching to Trametinib can benefit patients who have not responded to other treatments like Vemurafenib.
Study on Vemurafenib for Children with BRAF Mutation-Resistant Histiocytosis
This trial investigates the use of Vemurafenib in children whose histiocytosis has not responded to standard treatments and who have a specific genetic change called a BRAF mutation. The study seeks to determine the optimal dose and duration of treatment for young patients.
Who can participate: Children and teenagers with histiocytosis who have a confirmed BRAF mutation in their tumor tissue or blood are eligible. The disease must have failed to respond to previous treatments, which may include progression during initial therapy with Vinblastine and prednisolone, recurrence after initial response, multiple relapses, or disease that has returned after previous Vemurafenib treatment. Patients showing signs of brain-related complications on MRI scans may also be included. The patient or guardian must provide informed consent, and those of childbearing age must use effective contraception during treatment and for at least one year after stopping. Participants must be enrolled in the HISTIOGEN trial, and both male and female patients are welcome.
Who cannot participate: Patients who do not have histiocytic cell proliferation, those without a BRAF mutation, adults, and those whose disease has responded to standard treatments cannot participate.
What the trial aims to achieve: The study will evaluate the safety, effectiveness, and tolerability of Vemurafenib in children with BRAF-positive histiocytosis. Researchers aim to identify a dosage that provides similar benefits in children as seen in adults. The trial will monitor event-free survival, progression-free survival, overall survival, and how often the disease returns after two years. Regular health monitoring, including vital signs, laboratory tests, heart function assessments, and imaging studies, will ensure patient safety throughout the trial.
Investigational drug: Vemurafenib is a targeted therapy taken as a tablet. It works by inhibiting the mutated BRAF protein, which is involved in the growth and spread of cancer cells. By blocking this protein's activity, Vemurafenib aims to slow or stop the abnormal cell growth characteristic of BRAF-positive histiocytosis.
Summary
All three clinical trials are being conducted in Poland and focus on young patients with histiocytosis, including Langerhans cell sarcoma. The studies reflect a comprehensive approach to managing this rare condition, combining advanced diagnostic imaging with targeted therapies.
One trial explores the diagnostic potential of PET/CT imaging using Fludeoxyglucose (18F), which may help doctors better track disease activity and treatment response. The other two trials investigate targeted medications: Trametinib for patients without BRAF mutations or those who have not responded to Vemurafenib, and Vemurafenib itself for patients with confirmed BRAF mutations whose disease has proven resistant to conventional treatments.
A notable feature is that all therapeutic trials require participation in the HISTIOGEN trial, suggesting a coordinated research effort to advance understanding and treatment of this rare condition. The studies are expected to conclude by March 2026, with comprehensive monitoring of safety, effectiveness, and long-term outcomes. These trials represent important opportunities for young patients whose disease has not responded to standard treatments, offering access to innovative diagnostic tools and targeted therapies tailored to specific genetic characteristics of their condition.
