BRAF Negatif Çocuk Hastalarda Trametinib'in Süre ve Dozunun Optimizasyonu

Sade dilde özet

Bu çalışma, BRAF geninde mutasyon bulunmayan (ya da Vemurafenib tedavisi başarısız olan) dirençli histiyositoz (histiocytosis) olan çocuk hastalarda Trametinib ilacının uygulama süresi ve dozunu en uygun hale getirmeyi amaçlamaktadır. Tümör dokusunda ya da ctDNA'da BRAF mutasyonu olmayan ve önceki tedavileri başarısız olmuş, HISTIOGEN çalışmasına katılan hastalar katılabilmektedir. Çalışma Faz 2 aşamasında olup halen katılımcı almaktadır.

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

Faz 2 (Phase 2)

Durum

Katılımcı alıyor

Sponsor

Anna Raciborska

Müdahale

Trametinib

Başlangıç

01.04.2021

Tahmini bitiş

30.06.2027

Ülkeler

Poland

İlgili genler

Merkezler

• Warsaw, Poland

Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

Inclusion Criteria:

1. 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.
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

   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).
3. Signing of informed consent for trial participation (including for Trametinib treatment) according with current legal regulations.
4. 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.
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 Trametinib.
6. Persistent toxicity related to prior therapy, making it impossible to treat with Trametinib.
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.