Actively Recruiting

Phase 2
Age: 1Year - 18Years
All Genders
NCT04943198

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

Led by Anna Raciborska · Updated on 2026-03-27

25

Participants Needed

1

Research Sites

325 weeks

Total Duration

On this page

Sponsors

A

Anna Raciborska

Lead Sponsor

M

Maria Sklodowska-Curie National Research Institute of Oncology

Collaborating Sponsor

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 1Year - 18Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Presence of BRAF gene mutations in tumor tissues and/or circulating tumor DNA at any stage of treatment or follow-up
  • Failure of treatment defined by at least one of the following: progression on first or second line treatment including risk organ involvement; disease reactivation after initial response; third or subsequent disease reactivation; reactivation after vemurafenib therapy; signs of neurodegenerative disorder on CNS MRI
  • Signed informed consent for trial participation including vemurafenib treatment
  • Consent to use effective contraception during vemurafenib treatment and for at least 1 year after stopping in patients at puberty or sexual maturity
  • Participation in the HISTIOGEN trial
Not Eligible

You will not qualify if you...

  • Lack of inclusion criteria
  • Pregnancy or breastfeeding
  • Hypersensitivity to vemurafenib or any of its ingredients
  • Iritis, uveitis, or obstruction of retinal veins
  • Concurrent treatment with drugs that may interact with vemurafenib
  • Persistent toxicity from prior therapy preventing vemurafenib treatment
  • Diagnosis of other malignancies before study inclusion
  • Other acute or persistent disorders, behaviors, or abnormal lab results increasing risk or affecting study results as judged by the investigator

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Mother and Child Institute

Warsaw, Mazovian, Poland, 01-211

Actively Recruiting

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Research Team

K

Katarzyna Maleszewska

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

2

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