diffuse intrinsic pontine glioma
Conditions
Interventions
Trade Name: Mekinist 0,5 mg film-coated tablets
Pharmaceutical Form: Film-coated tablet
Trade Name: Rapamune 1 mg coated tablets
Pharmaceutical Form: Coated tablet
Trade Name: Rapamune 1mg/ml oral s
Sponsors
The Children's Memorial Health Institute
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age 3 to 18 years, inclusive. 2. Informed consent signed by parents or legal guardian of minor and by patient over 13 years of age) for the study diagnostic and therapeutic procedures, including molecular -based diagnostic and prognostic tests using NGS method. 3. Diagnosis of diffuse intrinsic pontine glioma (DIPG) based on clinical and radiological criteria 4. Histological diagnosis of glioma WHO grade III and IV confirmed by reference pathologist. Are the trial subjects under 18? yes Number of subjects for this age range: 100 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Any severe comorbid disease (eg kidney insufficiency, immunological deficiencies, HIV infection). 2. Clinically significant cardiovascular condition ( eg arrythmia) 3. Previous (< 5 yrs ) diagnosis of malignant neoplasm. 4. Active infection requiring systemic treatment. 5. Known allergy to the studied products. 6. Concomitant use of CYP3A or PgP inhibitors. 7. Radiological evidence of active intracranial hemorrhage (excluding receding post biopsy or focal bleeding). 8. Major surgery in the past 28 days
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The aim of the study is to develop the optimal treatment for patients with DIPG by identifying molecular markers important for therapy, adjusting the type of medicinal product to the indicated markers and assessing the safety and efficacy of selected drugs (sirolimus, trametinib) in the treatment of DIPG in children;Secondary Objective: 1. assessment of the efficacy and safety of selected drugs (sirolimus, trametinib) in the treatment of diffuse infiltrating ponsal gliomas in children; 2. to evaluate the safety and efficacy of the simultaneous irradiation and use of sirolimus; 3. adjusting the type of medicinal product to the indicated markers; 4. identification of molecular markers important for therapy; 5. creating a repository of genetic and clinical data of patients with DIPG; 6. development of the molecular profile of DIPG tumors.;Primary end point(s): 1.Overall survival (OS) from the time of diagnosis until last visit or death. 2.Safety assessment of sirolimus administered during radiotherapy and in combination with trametinib as adjunctive treatment after irradiation. ;Timepoint(s) of evaluation of this end point: Primary endpoints will be evaluated at 1,2,3 years from diagnosis. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Progression free survival from diagnosis to progression 2. Response rate assessed at time points specified in the protocol (MRI every 2 months from commnecement of systemic treatment) 3. Quality of life assessment (using PedQoL- pediatric quality of life inventory) at time points specified in the protocol. 4. Comparison of safety in 2 groups of patients; treated with sirolimus alone or in combination with trametinib. ;Timepoint(s) of evaluation of this end point: . | — |
Countries
Poland
Contacts
Public ContactBozenna Dembowska-Baginska
The Children's Memorial Health Institute
Outcome results
None listed