A comprehensive and targeted therapy approach in pediatric malignant pontine gliomas

Phase 2

Completed

Conditions: 10029211
brain stem glioma
malignant pontine glioma

Registration Number: NL-OMON44907

Lead Sponsor: Vrije Universiteit Medisch Centrum

Brief Summary: Trial ended prematurely

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 9

Inclusion Criteria

Patients with newly diagnosed, unresectable grade II-IV pontine glioma.
- Age between 3 and 18 years
- Willingness to perform a pregnancy test in females of child bearing age
- Written informed consent
- Platelet count * 100 x10 9/L (transfusion independent)
- Peripheral absolute neutrophil count (ANC) * 1.0 x10 9/L
- Direct bilirubin * 1.5 x upper limit of normal (ULN) for age
- SGPT (ALAT) < 5 x upper limit of normal (ULN) for age.
- Adequate Renal Function Defined As:
- Serum creatinine * 1.5 x upper limit of normal (ULN) for age;Phase B: patients with progressive disease after radiotherapy

Exclusion Criteria

- Pilocytic (grade 1) astrocytomas
- Cervicomedullary junction tumors
- Presence of diffuse leptomeningeal disease.
- Performance status (Lansky or Karnofsky score) of 40% or less
- Life expectancy of less than six weeks without further therapy
- Pregnant or breastfeeding
- Other contra-indications for chemotherapy
- Neurofibromatosis type I

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Phase A: Tolerability of gemcitabine at 3 dose levels (toxicity according to<br /><br>CTCAE-4)<br /><br><br /><br>Phase B: Tolerability of erlotinib and everolimus at two dose levels when added<br /><br>to bevacizumab-irinotecan (toxicity according to CTCAE-4)<br /><br></p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Phase A:<br /><br>Secondary endpoint: Clinical reponse rate, response rate on MRI according to<br /><br>the WHO-criteria and the median progression free survival<br /><br>Tertiary endpoints:<br /><br>- Quality of life, measured by standardised questionnaires (PedsQl).<br /><br>- The contribution of MRS to MRI in terms of predictors of survival or disease<br /><br>progression<br /><br><br /><br>Phase B:<br /><br>Secondary endpoint: Survival duration from time of disease progression<br /><br>Tertiary endpoints:<br /><br>- Response on MRI according to the WHO criteria<br /><br>- Quality of life, measured by standardised questionnaires (PedsQl).<br /><br>- Pharmacokinetics of erlotinib and everolimus<br /><br>- The contribution of MRS to MRI in terms of predictors of survival/disease<br /><br>progression</p><br>