Diffuse Intrinsic Pontine Glioma newly diagnosed in pediatric patients.
Conditions
Sponsors
Clínica Universidad de Navarra
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Informed consent OF PATIENT OR PARENTS 2. Patient must be, in the investigator opinion, able to comply with all the protocol procedures. 3. Age 1 - 18 years 4. Negative pregnant blood test in case of fertile women (A woman is considered of childbearing potential (WOCBP), i.e. fertile, following menarche and unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. 5. Patient newly diagnosed of DIPG in MRI 6. Lansky Performance Status = 70 before inclusion 7. Lesion considered by the investigator to be accessible for stereotactic biopsy. Lesion location will allow injection without entrance of virus in the ventricular system. 8. No previous treatment for DIPG Are the trial subjects under 18? yes Number of subjects for this age range: 12 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. Severe infections or intercurrent medical conditions including, but not limited to, severe renal, hepatic, heart or bone marrow failure, that, on investigator´s criteria, do not allow the inclusion. Patients must be afebrile at baseline [i.e., 3 times over upper normal laboratory level 9. Neutrophils < 1 x 109/L 10. Thrombocytes = 100 x 109/L 11. Hemoglobin < 9g/dl 13. Patients with Li-Fraumini Syndrome or with a known germ line deficit in the retinoblastoma gene or its related pathways. 14. Vaccinations of any kind within 30 days prior to DNX-2401 administration. 15. Transfusions or medications (G-CSF) to treat pancytopenia or other hematological conditions within 28 days of baseline.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the safety, tolerability and toxicity of DNX-2401 injected in the cerebellar peduncle in pediatric subjects with DIPG. The trial will look for hematologic and neurologic toxicity (NCI-CTCAE v 4.03).;Secondary Objective: •To determine Overall Survival at 12 months (OS12), complete/partial response in MRI, immune response induced by DNX-2401. •To measure quality of life (QoL) baseline assessment and any changes over time •To collect tumor and blood samples for futures molecular and immune studies.;Primary end point(s): To determine the safety, tolerability and toxicity of DNX-2401 injected in the cerebellar peduncle in pediatric subjects with DIPG. The trial will look for hematologic and neurologic toxicity.;Timepoint(s) of evaluation of this end point: For each individual patient the timepoint of evaluation will be two months after DNX.2401 injection, Fort he whole trial, could be 26-30 months (if the 12 patients are recruited along 24 months) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - To determine Overall Survival at 12 months (OS12), complete/partial response in MRI and immune response induced by DNX-2401. - To measure quality of life (QoL) baseline assessment and any changes over time. - To collect tumor and blood samples for futures molecular and immune studies.;Timepoint(s) of evaluation of this end point: 12 months after virus injection. | — |
Countries
Spain
Contacts
Public ContactUCEC
Clínica Universidad de Navarra
Outcome results
None listed