Optimizing vemurafenib treatment for children with refractory histiocytosis
Optimization of the Time and Dosage of Vemurafenib in BRAF Positive Juvenile Patients With Refractory Histiocytosis
This study is testing the best way to use the medication vemurafenib for children with hard-to-treat histiocytosis who have a specific gene mutation.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 25 (estimated) |
| Ages | 1 Year to 18 Years |
| Sex | All |
| Sponsor | Institute of Mother and Child, Warsaw, Poland Academic / other |
| Locations | 1 site (Warsaw, Mazovian) |
| Trial ID | NCT04943198 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to optimize the time and dosage of vemurafenib in juvenile patients with histiocytosis that is resistant to conventional therapies and has a confirmed BRAF gene mutation. It is a prospective, interventional, open, randomized, single-center trial that is part of the POLHISTIO project, which focuses on improving diagnosis and treatment for these patients. The study will evaluate the frequency of mutations, compare molecular test results with clinical data, and assess the prognostic value of molecular analysis. Patients will be recruited from across Poland.
Who should consider this trial
Good fit: Ideal candidates for this study are juvenile patients with histiocytosis who have a confirmed BRAF gene mutation and have failed previous treatments.
Not a fit: Patients without a BRAF gene mutation or those who have not experienced treatment failure may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a more effective treatment protocol for children suffering from refractory histiocytosis, potentially improving their outcomes.
How similar studies have performed: Other studies have shown promise in using targeted therapies for BRAF mutations, suggesting that this approach may be beneficial.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. 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. 2. Failure of the treatment (at least one of below needs to apply in order for this requirement to be satisfied): 1. 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 2. 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 3. Third or subsequent reactivation of disease with or without risk organ involvement, or 4. Reactivation of disease after Vemurafenib therapy has been completed, or 5. The appearance of signs of neurodegenerative disorder (ND) in MRI of the central nervous system (CNS). 3. Signing of informed consent for trial participation (including for Vemurafenib treatment) according with current legal regulations. 4. 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. 5. Participation in HISTIOGEN trial. Exclusion Criteria: 1. Lack of inclusion criteria. 2. Pregnancy and breastfeeding . 3. Hypersensitivity to the study drug or any of its ingredients. 4. Iritis, uveitis, obstruction of the retinal veins. 5. Simultaneous treatment with other drugs which might interact with Vemurafenib. 6. Persistent toxicity related to prior therapy, making it impossible to treat with Vemurafenib. 7. Diagnosis of other malignancies before study inclusion. 8. 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.
Where this trial is running
Warsaw, Mazovian
- Mother and Child Institute — Warsaw, Mazovian, Poland (Recruiting)
Study contacts
- Principal investigator: Anna Raciborska — Mother and Child Institute
- Study coordinator: Katarzyna Maleszewska
- Email: klinika.onkologii@imid.med.pl
- Phone: +48 22 32 77 205
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.