Optimization of the Time and Dosage of Trametinib in BRAF Negative Juvenile Patients

Recruiting now · Phase 2

Conditions studied: Histiocytosis

In brief

Prospective, interventional, open, randomized, single-center, non-commercial clinical trial to optimize treatment and dosage of trametinib in juvenile patients with histiocytosis resistant to conventional therapy and without the BRAF gene mutation or after the failure of vemurafenib treatment.

Key facts

Study ID

NCT04943224

Run by

Anna Raciborska

People needed

12

Starts

2021-04-01

Expected to finish

2027-06-30

Last updated by the study team

2026-03-27

Who can join

Age: 1 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Lack of mutations in the BRAF gene in tumor tissues and/or circulating tumor DNA (ctDNA) at any stage of treatment or follow-up, or failure of Vemurafenib treatment in BRAF positive patients.
• 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
• d. Progression during Vemurafenib therapy, or e. Reactivation of disease after Vemurafenib therapy has been completed, or f. 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 Trametinib treatment) according with current legal regulations.
• Consent to the use of effective contraception throughout the Trametinib administration period and a minimum of 1 year after discontinuation in patients at puberty and sexual maturity.
• Participation in HISTIOGEN trial.

You may not qualify 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 Trametinib.
• Persistent toxicity related to prior therapy, making it impossible to treat with Trametinib.
• 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.

Where it is running

• Mother and Child Institute — Warsaw, Mazovian, Poland (enrolling)

Full record on ClinicalTrials.gov

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