Prospective, interventional, open, randomized, single-center, non-commercial clinical trial to optimize treatment and dosage of vemurafenib in juvenile patients with histiocytosis resistant to conventional therapy and in whom the BRAF gene mutation has been found.
The presence of mutations in the BRAF gene in tumor tissues and/or in circulating tumor DNA (ctDNA) at any stage of treatment or follow-up.
Progression on the I and/or II line treatment, including at least one risk organ; prior treatment should include a minimum of 6 weeks of weekly Vinblastine with a minimum of 28 days prednisolone or minimum 2 cycles of Cytosine Arabinoside in 4-day cycles and/or Cladribine in 5-day cycles as a 2nd line treatment, minimum 2 cycles, or other second-line treatment or
Disease reactivation after an initial response to treatment with Vimblastine and prednisolone as the first line and/or no response to second line treatment using one of two drugs: Cytosine Arabinoside in 4-day cycles and/or Cladribine in 5-day cycles, minimum 2 cycles, or other I/ II line treatment or occurrence of involvement of at least one risk organ or
Third or subsequent reactivation of disease with or without risk organ involvement, or
Lack of inclusion criteria.
Pregnancy and breastfeeding .
Hypersensitivity to the study drug or any of its ingredients.
Iritis, uveitis, obstruction of the retinal veins.
Single group
Vemurafenib
Drug20 mg/kg/day
event-free survival
Event-free survival (EFS) was defined as the time interval from the date of diagnosis to the date of disease progression, recurrence, second malignancy, death or to date of last follow-up for patients without events.
Molecular relapse (in ct DNA)
Molecular relapse was defined as the time interval from the date of BRAF negativization to the date of positive results of BRAF mutation
Time to negative mutation test results (in ct DNA)
Time to negative mutation test results (in ct DNA) was defined as the time interval from the date of positive BRAF mutation to the date of negative results of BRAF mutation
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Anna Raciborska
Lead sponsor
Institute of Mother and Child, Warsaw, Poland
Sponsor institution
Maria Sklodowska-Curie National Research Institute of Oncology
Collaborator
Łukasiewicz Research Network
Collaborator
Wroclaw University of Environmental and Life Sciences
Collaborator
