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

Who is this study for? Children and young adults up to age 18 with histiocytosis
What treatments are being studied? Vemurafenib
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 1
Maximum Age: 18
Healthy Volunteers: f
View:

• 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.

• 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/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

⁃ Reactivation of disease after Vemurafenib therapy has been completed, or

⁃ The appearance of signs of neurodegenerative disorder (ND) in MRI of the central nervous system (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.

Locations
Other Locations
Poland
Mother and Child Institute
RECRUITING
Warsaw
Contact Information
Primary
Katarzyna Maleszewska
klinika.onkologii@imid.med.pl
+48 22 32 77 205
Time Frame
Start Date: 2021-04-01
Estimated Completion Date: 2026-03-30
Participants
Target number of participants: 25
Treatments
Experimental: R1 time of vemurafenib treatment
vemurafenib will be given to 6 months after BRAF negativization
Experimental: R2 time of vemurafenib treatment
vemurafenib will be given to 12 months after BRAF negativization
Related Therapeutic Areas
Sponsors
Collaborators: Wrocław University of Environmental and Life Sciences, Maria Sklodowska-Curie National Research Institute of Oncology, Łukasiewicz Research Network
Leads: Anna Raciborska

This content was sourced from clinicaltrials.gov