Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00411619
• Date of registration: 12/12/2006
• Prospective Registration: Yes
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex
• Scientific title: Everolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis Complex
• Date of first enrolment: January 2007
• Target sample size: 28
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00411619
• Study type: Interventional
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: David N Franz, M.D.
• Affiliation: Children's Hospital Medical Center, Cincinnati

Key inclusion & exclusion criteria

Inclusion Criteria:

- Age three years and older

- If female and of child bearing potential, documentation of negative pregnancy test
prior to enrollment. In the extension phase, all females of child bearing potential
will be required to take monthly home pregnancy tests and record results on a provided
diary card.

- Clinically definite diagnosis of tuberous sclerosis (modified Gomez criteria or
positive genetic test)

- Presence of giant cell astrocytoma as defined by imaging characteristics and serial
increase in size of lesion on 2 or more MRI scans

- Adequate renal function (creatinine < 1.5 mg/dl)

Exclusion Criteria:

- Serious intercurrent medical illness or other uncontrolled medical disease which could
compromise participation in the study

- Significant hematologic or hepatic abnormality

- Continuous requirement for supplemental oxygen

- Intercurrent infection at initiation of everolimus

- Embolization of angiomyolipoma within one month; any other recent surgery within 2
months of initiation of everolimus

- Pregnant or lactating women

- Use of an investigational drug within the last 30 days

- Must be adequately recovered from the acute toxicities of any prior therapy

- Clinical evidence of impending herniation or focal neurologic deficit related to the
subject's astrocytoma

- Unwilling or unable to use highly effective contraception



Age minimum: 3 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Subependymal Giant Cell Astrocytoma

Tuberous Sclerosis

Intervention(s)

Drug: Everolimus

Primary Outcome(s)

Number With Observed Adverse Side Effects [Time Frame: During the entire study]

Secondary Outcome(s)

Overall Reduction in SEGA Tumor Volume. [Time Frame: During the entire study]

Secondary ID(s)

CCHMC IRB# 06-07-50

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Novartis

Results

• Results available: Yes
• Date Posted: 20/10/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00411619