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

ConditionHistiocytosis
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-18
SponsorAnna Raciborska

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

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

Signing of informed consent for trial participation (including for Trametinib treatment) according with current legal regulations.

Disqualifiers

Lack of inclusion criteria.

Pregnancy and breastfeeding .

Hypersensitivity to the study drug or any of its ingredients.

Iritis, uveitis, obstruction of the retinal veins.

Trial design

Design model

Single group

Treatments tested in this trial

  • Trametinib

    Drug

    \< 6 lat 0,032mg/kg, ≥ 6 lat 0,025mg/kg,

Treatment groups

12 Participants
are divided into 2 treatment groups
Group A: R1 time of trametinib treatmentExperimental treatment 1 intervention
Group B: R2 time of trametinib treatmentExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Event-free survival (EFS)

Event-free survival (EFS) was defined as the time interval from the date of diagnosis to the date of disease progression, recurrence, second malignancy, death or to date of last follow-up for patients without events.

Time frame
2 years

Secondary outcomes

1

Molecular relapse (in ct DNA)

Molecular relapse was defined as the time interval from the date of any mutation negativization to the date of positive results of any mutation

Time frame
2 years
2

Time to negative mutation test results (in ct DNA)

Time to negative mutation test results (in ct DNA) was defined as the time interval from the date of positive mutation to the date of negative results of any mutation

Time frame
2 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Anna Raciborska

Lead sponsor

Institute of Mother and Child, Warsaw, Poland

Sponsor institution

Maria Sklodowska-Curie National Research Institute of Oncology

Collaborator

Łukasiewicz Research Network

Collaborator

Wroclaw University of Environmental and Life Sciences

Collaborator

Source ClinicalTrials.gov
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