Optimization of the Time and Dosage of Vemurafenib in BRAF Positive Juvenile Patients With Refractory Histiocytosis

  • STATUS
    Recruiting
  • End date
    Jun 23, 2026
  • participants needed
    25
  • sponsor
    Anna Raciborska
Updated on 8 July 2021

Summary

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.

Description

BRAVO clinical study is part of the POL HISTIO project. The POL HISTIO project is a non-commercial clinical trial aimed at optimizing the diagnosis and treatment of juvenile patients with histiocytosis. The project objectives are defined as follows: 1) to estimate the nature and frequency of mutations in patients with histiocytosis in both tumor tissues and free-circulating DNA; 2) to compare molecular test results with clinical data; 3) to evaluate the diagnostic usefulness of the status of molecular analysis (MRD) as a prognostic factor compared with other recognized factors; 4) in the case of failure of conventional therapy - to modify treatment and to apply targeted treatment, based on molecular status of gene mutation. The project is intended to include patients from all over Poland.

Details
Condition Histiocytosis
Treatment Vemurafenib
Clinical Study IdentifierNCT04943198
SponsorAnna Raciborska
Last Modified on8 July 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

The presence of mutations in the BRAF gene in tumor tissues and/or in ctDNA at any stage of treatment or follow-up
Failure of the treatment (at least one of below needs to apply in order for this requirement to be satisfied)
Progression on the I and II line treatment, and involving of a minimum one risk organ; previous 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 treatment of 2 line, or
Disease reactivation after an initial response to treatment with Vimblastine and prednisolone as the first line and no response to treatment of the second line using a minimum of 2 cycles of one of two drugs: Cytosine Arabinoside in 4-day cycles and/or Cladribine in 5-day cycles as a treatment of 2 line and the involving of a minimum one risk organ, or
Third or subsequent reactivation of disease with or without risk organ involvement, or
Reactivation of disease after Vemurafenib therapy has been completed, or
The appearance of signs of neurodegenerative disorder (ND) in MRI of the CNS
Signing of informed consent for trial participation (including for Vemurafenib treatment) according with current legal regulations
Consent to the use of effective contraception throughout the Vemurafenib administration period and a minimum of 1 year after discontinuation in patients at puberty and sexual maturity
Participation in HISTIOGEN trial

Exclusion Criteria

Lack of inclusion criteria
Pregnancy and breastfeeding
Hypersensitivity to the study drug or any of its ingredients
Iritis, uveitis, obstruction of the retinal veins
Simultaneous treatment with other drugs which might interact with Vemurafenib
Persistent toxicity related to prior therapy, making it impossible to treat with Vemurafenib
Diagnosis of other malignancies before study inclusion
Other acute or persistent disorders, behaviors or abnormal laboratory test results, which might increase the risk related to the participation in this clinical trial or to taking the study drug, or which might influence the interpretation of the study results, or which, in the investigator's opinion, disqualify a patient from participating in the trial
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