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| Diagnosis | Plexiform Neurofibroma, Neurofibromatosis, LGG, Low Grade Glioma, LCH, Langerhans Cell Histiocytosis, other solid tumors and brain tumors | Study Status | Completed |
| Phase | I/II |
| Age | Child - (1 Month to 17 Years) | Randomisation | NO |
| Line of treatment | First line treatment, Disease relapse or progression |
| Routes of Treatment Administration | Trametinib: oral (capsules, tablets or suspension)
Dabrafenib: oral (capsules, tablets or suspension)
|
| Last Posted Update | 2021-11-03 |
| ClinicalTrials.gov # | NCT02124772 |
International Sponsor
Novartis PharmaceuticalsPrincipal Investigators for Canadian Sites
The Hospital for Sick Children – Dr. James WhitlockCentres
Study Description
Some diseases such as cancer with BRAF mutations, low grade gliomas and plexiform neurofibroma in patients with neurofibromatosis type 1 [NF-1], or Langerhans cell histocytosis [LCH]) have been shown to have molecular dysfunction called MAPK pathway dysregulation which favors tumour development. The MAPK pathway is involved in cell growth, differentiation, inflammation and apoptosis.Trametinib is an inhibitor of components of the MAPK pathway called MEK1 and MEK2. Drabafenib is a small molecule that inhibits the MAPK pathway in BRAF mutated cells by blocking the BRAF serine-threonine kinase. Both are expected to slow or stop the growth of cancer cells.
The aim of this phase I/II study is to evaluate the dose, safety, tolerability, antitumor activity and other pharmacologic characteristics of Trametinib alone or with Dabrafenib in children with refractory or relapsed solid cancer or disease with BRAF mutations for which no standard therapy is available or for which the subject is not eligible for the existing therapy.
The study is currently open for LCH and neuroblastoma only.
Inclusion Criteria
- Male or female between one month and <18 years of age (inclusive) at the time of signing consent
- Must have a disease that is relapsed/refractory to all potentially curative standard treatment regimens or must have a current disease for which there is no known curative therapy, or therapy proven to prolong survival with an acceptable quality of life.
- The subject's disease (i.e. cancer, neurofibromatosis type 1 [NF-1] with plexiform neurofibroma [PN], or Langerhans cell histocytosis [LCH]) must have relapsed after or failed to respond to frontline curative therapy or there must not be other potentially curative treatment options available. Curative therapy may include surgery, radiation therapy, chemotherapy, or any combination of these modalities
- Able to swallow and retain enterally administered medication
Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.
