Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Brief Title

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Official Title

A Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Brief Summary

      This study will evaluate the safety and efficacy of RAD001 in treating patients with
      Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic
      Lymphangioleiomyomatosis.
    

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Angiomyolipoma Response Rate as Per Central Radiology Review

Secondary Outcome

 Time to Angiomyolipoma Progression as Per Central Radiology Review

Condition

Tuberous Sclerosis Complex (TSC)

Intervention

Everolimus (RAD001)

Study Arms / Comparison Groups

 Everolimus

Description:  Study drug was given by continuous oral daily dosing of two 5 mg tablets.

Publications

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

Recruitment Status

Drug

Estimated Enrollment

118

Start Date

April 2009

Completion Date

November 2015

Primary Completion Date

June 2011

Eligibility Criteria

        Inclusion Criteria:

          -  Male or Female 18 years or older

          -  Clinically definite diagnosis of Tuberous Sclerosis Complex according to the modified
             Gomez criteria or sporadic LAM (biopsy-proven or compatible chest CT scan)

          -  Clinically definite diagnosis of renal angiomyolipoma

          -  At least one Angiomyolipoma of ≥ 3 cm in its longest diameter using CT or MRI

          -  Females of child bearing potential must use birth control and have documentation of
             negative pregnancy test

          -  Written informed consent according to local guidelines

        Exclusion Criteria:

          -  Recent heart attack, cardiac related chest pain or stroke

          -  Severely impaired lung function

          -  Bleeding related to angiomyolipoma or embolization during 6 months prior to
             randomization

          -  Clinically significant chylous ascites

          -  Clinically significant hematological or hepatic abnormality

          -  Severe liver dysfunction

          -  Severe kidney dysfunction

          -  Pregnancy or breast feeding

          -  Current infection

          -  History of organ transplant

          -  Surgery within two months prior to study enrollment

          -  Prior therapy with a medication in the same class as Everolimus

          -  Recent use of an investigational drug

          -  Bleeding diathesis or on oral anti-vitamin K medication

          -  Uncontrolled high cholesterol

          -  Uncontrolled diabetes

          -  HIV

          -  Inability to attend scheduled clinic visits

          -  Patients with metal implants thus prohibiting MRI evaluations

          -  Angiomyolipoma which requires surgery at the time of randomization

          -  History of malignancy

          -  Severe or uncontrolled medical conditions which would cause an unacceptable safety
             risk or compromise compliance with the protocol
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Novartis Pharmaceuticals, , 

Location Countries

Canada

Location Countries

Canada

NCT ID

NCT00790400

Organization ID

CRAD001M2302

Secondary IDs

2008-002113-48

Responsible Party

Sponsor

Study Sponsor

Novartis Pharmaceuticals

Study Sponsor

Novartis Pharmaceuticals, Study Director, Novartis Pharmaceuticals

Verification Date

January 2017