Maintenance therapy with Everolimus in patients with tuberous sclerosis successfully treated for subependymal giant cell astrocytoma

Phase 4

Conditions: Q85.1
D33.0
Tuberous sclerosis
Brain, supratentorial

Registration Number: DRKS00005584

Lead Sponsor: Medical University of Lodz

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Complete

Sex: All

Target Recruitment: 12

Inclusion Criteria

Diagnosis of Tuberous sclerosis and supependymal giant astrocytoma.
At least 12 months everolimus treatment resulting in reduction (more than 30% decrease of tumor volume) or stabilization (less than 20% increase in tumor volume) of SEGA volume.
No signs of increased intracranial pressure/no hydrocephalus in MRI
Signed informed consent to the trial.

Exclusion Criteria

No diagnosis of TSC
Age > 20 years
Progression (more than 20% increase of tumor volume) of SEGA volume during everolimus treatment in standard dose.
Signs of increased intracranial pressure/hydrocephalus in MRI during everolimus treatment in standard dose.
No signed informed consent to the trial.

Study & Design

Study Type: interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Proportion of patients with stable or acceptable increase of SEGA volume, defined as no changes in the total volume of all target SEGA or increase less than 50% relative to baseline and no greater than volume before start of everolimus treatment, no new lesions of 1 cm or greater in diameter, and new or worsening hydrocephalus. Magnetic resonance evaluation will be performed at the study entery and after 3, 6, 12, 18, 24 months of the study.

Secondary Outcome Measures

Name	Time	Method
Time to progression of subependymal giant cel astrocytoma.<br>Number, type and severity of complications of everolimus treatment in reduced dose. Clinical evaluation of the progression and presence of the treatment-related side effects will be performed during monthly follow-u visits.<br>