Ongoing Clinical Trials for Hairy Cell Leukaemia
There are currently 3 clinical trials investigating new treatments for hairy cell leukaemia, a rare blood cancer affecting white blood cells. These studies are exploring chemotherapy-free alternatives and advanced immunotherapies across multiple European countries, offering hope to patients who have not yet been treated or whose disease has returned after previous treatment.
Clinical trial locations
- Austria
- France
- Germany
- Italy
- Study of Obinutuzumab, Vemurafenib, and Cobimetinib for Hairy Cell Leukemia in Patients Previously Treated with Purine Analogs or Unfit for Chemotherapy
- Study on Brexucabtagene Autoleucel for Adults with Relapsed/Refractory Hairy Cell Leukemia
- Study of Vemurafenib and Rituximab for Untreated Hairy Cell Leukemia Patients
- Netherlands
- Spain
- Sweden
Study of Vemurafenib and Rituximab for Untreated Hairy Cell Leukemia Patients
This trial is testing a new chemotherapy-free treatment approach for patients who have never received treatment for hairy cell leukaemia. The study combines two medications, vemurafenib and rituximab, and compares them with the standard treatment, cladribine.
Who can participate: Adults aged 18 and older with newly diagnosed hairy cell leukaemia who need treatment due to low blood counts, an enlarged spleen, or other organ problems. Patients must have a specific genetic mutation called BRAF-V600E and be able to swallow tablets. Good kidney and liver function are required, and patients must not have serious uncontrolled infections. Women who can become pregnant must use effective birth control during and after the study.
Who cannot participate: Patients who have already received treatment for the condition, those without a confirmed diagnosis, and individuals considered part of vulnerable populations who may have difficulty providing informed consent.
Study focus: The main goal is to determine whether the combination of vemurafenib and rituximab is as effective as standard chemotherapy while being less toxic. Researchers will monitor how well the cancer responds to treatment and how long it takes for the disease to return, if at all. The trial includes regular assessments over several months, with additional rituximab doses given if needed based on remaining disease activity.
Medications being tested: Vemurafenib is taken as an oral tablet and works by targeting a specific protein in cancer cells to stop their growth. Rituximab is given through an intravenous infusion and is a type of antibody therapy that helps the immune system destroy cancer cells. Cladribine, the standard chemotherapy drug used for comparison, interferes with cancer cell growth.
Study of Obinutuzumab, Vemurafenib, and Cobimetinib for Hairy Cell Leukemia in Patients Previously Treated with Purine Analogs or Unfit for Chemotherapy
This study is designed for patients who have already been treated with certain drugs called purine analogs or who are not suitable candidates for chemotherapy. It tests a combination of three medications: obinutuzumab, vemurafenib, and cobimetinib.
Who can participate: Adults aged 18 and older with confirmed hairy cell leukaemia and the BRAF-V600E mutation who need treatment based on low blood counts, an enlarged symptomatic spleen, or significant involvement of other organs. Patients must have completed their previous treatment at least 12 weeks before joining the study and have recovered from any side effects. They must have a performance status score of 0 to 2, meaning they can take care of themselves even if unable to work. Fertile men and women must use effective contraception during and after treatment.
Who cannot participate: Patients without the BRAF-V600E mutation, those with allergies to the study drugs, pregnant or breastfeeding women, individuals with other serious health conditions that might interfere with treatment, patients currently in another clinical trial, those with recent or severe heart problems, and individuals with severe liver or kidney problems.
Study focus: The trial aims to understand how effective this three-drug combination is in fighting the leukaemia. Researchers will evaluate the depth of anti-leukemic activity, time to response, relapse-free survival, and overall survival. The treatment is given in a step-wise manner with regular monitoring for side effects and treatment response.
Medications being tested: Obinutuzumab is given intravenously and is an antibody therapy that targets specific proteins on cancer cells to help the immune system destroy them. Vemurafenib is taken orally and blocks a mutation called BRAF-V600E to slow cancer cell growth. Cobimetinib is also taken orally and works by blocking a protein called MEK, enhancing the effects of vemurafenib by disrupting the pathway that helps cancer cells grow.
Study on Brexucabtagene Autoleucel for Adults with Relapsed/Refractory Hairy Cell Leukemia
This trial tests an advanced cell therapy called brexucabtagene autoleucel for patients whose disease has returned after treatment or has not responded to previous therapies. This innovative treatment uses a patient’s own modified immune cells to fight cancer.
Who can participate: Adults aged 18 and older with confirmed hairy cell leukaemia who need treatment due to low blood counts, symptomatic enlarged spleen, or symptomatic swollen lymph nodes. Patients must have received at least two prior therapies, including a purine nucleoside analog treatment. Any side effects from previous treatments must have improved to a mild level. Patients must have a performance status score of 0 or 1 and adequate blood and organ function. Those able to have children must agree to use specified contraception methods.
Who cannot participate: Patients who have not been diagnosed with relapsed or refractory hairy cell leukaemia, those outside the specified age range, individuals not part of the specified clinical trial group, and those unable to provide informed consent or considered part of vulnerable populations.
Study focus: The trial evaluates how effective brexucabtagene autoleucel is in helping patients with relapsed or refractory disease. It monitors response rates, duration of response, overall survival, and time before additional treatment might be needed. The study assesses both safety and effectiveness of this cell therapy approach.
Medications being tested: Brexucabtagene autoleucel is the main treatment, which involves taking a patient’s own white blood cells, modifying them in a laboratory to better recognize and attack cancer cells, and then infusing them back into the patient. Supporting medications include fludarabine and cyclophosphamide, given before the main treatment to prepare the body, and methylprednisolone and dexamethasone, which are corticosteroids used to help manage side effects and reduce inflammation.
Summary
The three ongoing clinical trials for hairy cell leukaemia represent different treatment strategies for various patient groups. Two trials are being conducted in Italy, focusing on chemotherapy-free alternatives for both newly diagnosed and previously treated patients. A third multi-country trial across seven European nations is testing an advanced cell therapy for patients with relapsed or refractory disease.
A notable trend is the focus on targeted therapies that block the BRAF-V600E mutation, which is present in many patients with this condition. Two trials use vemurafenib to target this mutation, with one combining it with rituximab for untreated patients and another adding cobimetinib and obinutuzumab for previously treated patients. The third trial takes a different approach using brexucabtagene autoleucel, a personalised cell therapy that modifies a patient’s own immune cells.
These studies reflect a shift away from traditional chemotherapy towards more targeted and personalised treatment approaches. Patients interested in participating should discuss eligibility requirements with their healthcare providers, as each trial has specific criteria regarding prior treatments, genetic mutations, and overall health status.
