Ongoing Clinical Trials for Langerhans’ Cell Histiocytosis
There are currently 4 clinical trials investigating new treatments and diagnostic approaches for Langerhans’ cell histiocytosis, a rare condition involving abnormal growth of immune cells. These studies are taking place across multiple countries in Europe and focus on various medications and imaging techniques to improve patient outcomes, particularly in children and adolescents.
Clinical trial locations
- Austria
- Belgium
- Czechia
- Denmark
- Greece
- Ireland
- Italy
- Netherlands
- Norway
- Poland
- Study on Langerhans Cell Histiocytosis Treatment in Children and Adolescents Using Cladribine, Prednisolone, and Cytarabine
- Study on the Use of Fludeoxyglucose (18F) in PET/CT Scans for Young Patients with Histiocytosis
- Study on Trametinib Dimethyl Sulfoxide for Children with Refractory Histiocytosis Not Responding to Conventional Treatment
- Study on Vemurafenib for Children with BRAF Mutation-Resistant Histiocytosis
- Spain
- Sweden
Study on the Use of Fludeoxyglucose (18F) in PET/CT Scans for Young Patients with Histiocytosis
This trial is investigating the use of a specialized imaging technique called PET/CT scanning to better understand and monitor the condition in young patients. The scan uses a radioactive substance called Fludeoxyglucose (18F), which is a type of sugar that helps doctors see areas of high activity in the body.
Who can participate: The study is open to patients under 18 years of age who have confirmed or suspected histiocytosis based on previous test results. The patient or their guardian must sign an informed consent form agreeing to participate in the study.
Who cannot participate: There are no specific exclusion criteria listed for this study.
What the trial focuses on: The main goal is to determine whether using Fludeoxyglucose (18F) in PET/CT imaging is safe and beneficial for young patients with this condition. The study will assess how well the imaging helps doctors understand the disease at a molecular level and monitor its progression over time. Researchers will track how long patients remain free from disease worsening and will monitor any side effects that occur during the study.
Investigational substance: Fludeoxyglucose (18F-FDG) is a radioactive sugar administered through an injection into a vein. It is particularly useful for identifying areas of high metabolic activity in the body, which can indicate the presence of disease. This imaging agent helps doctors visualize what is happening inside the body at a cellular level.
Study on Trametinib Dimethyl Sulfoxide for Children with Refractory Histiocytosis Not Responding to Conventional Treatment
This trial is testing a medication called Trametinib for children whose condition has not improved with standard treatments. Trametinib is taken as a tablet and aims to help manage the disease when other therapies have failed.
Who can participate: The study is for children and teenagers with histiocytic cell proliferation who do not have mutations in the BRAF gene, or who have tried Vemurafenib treatment without success. Patients must have tried other treatments that did not work, including medicines like Vinblastine and prednisolone, or other medications such as Cytosine Arabinoside or Cladribine. The patient or guardian must sign an informed consent form, and if the patient is old enough to have children, they must agree to use effective birth control during the study and for at least one year afterward. Participants must also be enrolled in another study called the HISTIOGEN trial.
Who cannot participate: The study is only for patients with histiocytic cell proliferation who meet the specific criteria listed above. It is not open to patients with different conditions, adults, or those who have not previously tried the required treatments.
What the trial focuses on: The main goal is to evaluate how safe and effective Trametinib is for children with treatment-resistant disease. Researchers will determine the best dosage and duration of treatment for this age group. The study will monitor how long patients live without the disease getting worse, track overall survival rates, and record any side effects. Participants will receive regular health checks, including laboratory tests and heart monitoring, to ensure their safety throughout the trial.
Investigational drug: Trametinib is a medication that works by inhibiting a specific protein in the cell signaling pathway, which helps slow down or stop the growth of abnormal cells. It is classified as a MEK inhibitor, a type of targeted therapy.
Study on Vemurafenib for Children with BRAF Mutation-Resistant Histiocytosis
This trial is studying Vemurafenib, a medication taken as a tablet, for children whose condition involves a specific genetic change called the BRAF mutation and who have not responded to other treatments.
Who can participate: The study is for patients who have a mutation in the BRAF gene found in their tumor tissue or blood. They must have experienced treatment failure, which can include disease worsening during initial treatment with drugs like Vinblastine and prednisolone, disease returning after initial improvement, multiple relapses, or disease coming back after finishing Vemurafenib treatment. Patients showing signs of brain involvement on MRI scans may also be eligible. The patient or guardian must sign an informed consent form, and those of childbearing age must agree to use effective birth control during treatment and for at least one year after stopping. Participants must be enrolled in the HISTIOGEN trial.
Who cannot participate: The study is specifically for children and young people with BRAF-positive, treatment-resistant histiocytic cell proliferation. Those who do not meet these criteria cannot participate.
What the trial focuses on: The main objective is to evaluate the safety, effectiveness, and tolerability of Vemurafenib in children with the BRAF mutation whose disease has not responded to standard treatments. Researchers aim to find a dose for children that provides similar benefits to those seen in adults. The study will monitor how well the drug prevents disease progression, how long patients live without worsening, and how often the disease returns after two years. Regular health checks including vital signs monitoring, laboratory tests, and imaging will be conducted throughout the trial.
Investigational drug: Vemurafenib works by targeting and inhibiting the mutated BRAF protein, which is involved in the growth and spread of abnormal cells. It is classified as a BRAF inhibitor, a type of targeted therapy designed to specifically attack cells with this genetic mutation.
Study on Langerhans Cell Histiocytosis Treatment in Children and Adolescents Using Cladribine, Prednisolone, and Cytarabine
This comprehensive trial is investigating various treatment approaches for Langerhans Cell Histiocytosis in young patients. The study uses multiple medications in different combinations to find the most effective ways to treat the condition and prevent it from returning.
Who can participate: The study is open to patients younger than 18 years old who have a confirmed diagnosis of Langerhans Cell Histiocytosis based on tissue sample testing. Participants must not have received any previous treatments that affect the whole body, and they or their guardians must sign an informed consent form. Patients must also meet specific criteria for the particular treatment group they are being considered for.
Who cannot participate: The study excludes patients who have not responded to first-line therapy for the condition when risk organs are involved, those with medical conditions that might interfere with the study treatment, those unable to follow the study procedures, pregnant or breastfeeding women, patients with other significant medical conditions that could affect results, those currently in another clinical trial, patients with allergies to study medications, those with severe liver or kidney disease, and patients with a history of drug or alcohol abuse.
What the trial focuses on: The study has several main goals. For patients with multi-system disease, it aims to reduce the risk of death by quickly switching to alternative treatments if the first treatment does not work. It also explores whether extending or intensifying ongoing treatment can reduce the chances of disease returning and prevent long-term problems. For patients with single-system disease, the study investigates whether longer treatment can help prevent recurrence and reduce long-term complications. Participants will follow treatment plans lasting up to 24 months, with different combinations of medications used at various stages. The study will monitor disease resolution, prevention of reactivations, and minimization of long-term consequences.
Investigational drugs: The trial uses several medications:
- Prednisone: A corticosteroid that reduces inflammation and helps control the overactive immune system.
- Cytarabine: A chemotherapy drug that slows or stops the growth of abnormal cells by interfering with their DNA.
- Vincristine: A chemotherapy medication that stops cell growth by preventing cells from dividing.
- Indomethacin: An anti-inflammatory medication used to reduce inflammation and pain.
- 6-Mercaptopurine: An immune-suppressing medication used to maintain disease control and prevent reactivation.
- Methotrexate: A medication that slows cell growth and suppresses the immune system, used as part of long-term treatment strategy.
Summary
Currently, there are four ongoing clinical trials investigating treatments and diagnostic approaches for Langerhans’ cell histiocytosis. All trials focus exclusively on children and adolescents, reflecting the fact that this condition primarily affects younger patients.
Poland stands out as the most active location, hosting all four trials. One large multi-country trial is being conducted across twelve European nations, including Sweden, Denmark, Italy, Belgium, Spain, Greece, Norway, Czechia, Austria, Netherlands, Ireland, and Poland, demonstrating significant international collaboration in researching this rare condition.
The trials represent diverse approaches to managing the disease. Three studies focus on targeted drug therapies: Trametinib for patients without BRAF mutations or those who have not responded to Vemurafenib, Vemurafenib for patients with BRAF mutations, and a comprehensive multi-drug approach using Cladribine, Prednisolone, and Cytarabine among other medications. The fourth trial takes a different approach by investigating an imaging technique using Fludeoxyglucose (18F) in PET/CT scans to better understand and monitor the disease.
A common theme across the therapeutic trials is addressing treatment-resistant cases, with studies specifically targeting patients whose condition has not responded to conventional treatments. The research aims to find better ways to manage the disease, prevent recurrence, and minimize long-term complications for young patients living with this rare condition.
