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To check the safety, efficacy and tolerability of the drug vemurafenib, and to determine the duration of treatment in children with BRAF mutationresistant histiocytic cell hyperplasia resistant to conventional therapy.
EU CTIS ID: 2024-515805-25-00
What this study is testing
Optimalization of the time and dosage of Vemurafenib in BRAF positive juvenile patients with refractory histiocytosis.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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): a. 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 secondline treatment or b. 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 c. Third or subsequent reactivation of disease with or without risk organ involvement, or d. Reactivation of disease after Vemurafenib therapy has been completed, or e. 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.
You likely can't join if
- 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.
See the full eligibility criteria
Who can join
- 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): a. 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 secondline treatment or b. 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 c. Third or subsequent reactivation of disease with or without risk organ involvement, or d. Reactivation of disease after Vemurafenib therapy has been completed, or e. 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.
What rules you out
- 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.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.