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

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Brief Title

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

Official Title

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

Brief Summary

      The purpose of the study is to evaluate the safety and potential side effects of everolimus
      (an experimental drug) on a person with Tuberous Sclerosis Complex who also has been
      diagnosed with a brain tumor (astrocytoma)

      The hypothesis is that the drug will cause the tumor size to decrease, and may have
      beneficial activity separate from effects on tumors in patients.
    

Detailed Description

      Tuberous Sclerosis Complex (TSC)is a genetic disorder with a birth incidence of approximately
      one in six thousand. Five to twenty percent of patients with TSC will develop astrocytoma, a
      slowly progressive tumor. They grow and cause damage to surrounding brain tissue, blockage of
      spinal fluid (hydrocephalus), blindness, trouble walking, seizures, and brain damage. If
      untreated, they can be fatal. Standard treatment involves surgery to remove the tumor;
      however surgery may itself cause brain damage, bleeding, or infection, as well as other
      complications. Studies have shown that everolimus suppresses the chemicals that cause tumors
      to grow in tuberous sclerosis, and may cause them to shrink.

      The primary objective of this study is to find out the effects of everolimus on astrocytomas
      in a six month trial in patients with Tuberous Sclerosis who have been diagnosed with an
      astrocytoma
    

Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

Number With Observed Adverse Side Effects

Secondary Outcome

 Overall Reduction in SEGA Tumor Volume.

Condition

Tuberous Sclerosis

Intervention

Everolimus

Study Arms / Comparison Groups

 Everolimus
Description:  As this was a non-randomized, open-label, single arm study, all patients in the study received treatment with everolilmus

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

28

Start Date

January 2007

Completion Date

January 2014

Primary Completion Date

January 2014

Eligibility 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
      

Gender

All

Ages

3 Years - N/A

Accepts Healthy Volunteers

No

Contacts

David N Franz, M.D., , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00411619

Organization ID

CCHMC IRB# 06-07-50


Responsible Party

Sponsor

Study Sponsor

Children's Hospital Medical Center, Cincinnati

Collaborators

 Novartis

Study Sponsor

David N Franz, M.D., Principal Investigator, Children's Hospital Medical Center, Cincinnati


Verification Date

October 2014