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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

118 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-04-30

Study Completion Date

2015-11-30

Brief Summary

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

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Detailed Description

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Conditions

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Tuberous Sclerosis Complex (TSC) Lymphangioleiomyomatosis (LAM)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Everolimus

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

Group Type EXPERIMENTAL

Everolimus (RAD001)

Intervention Type DRUG

Everolimus is used in 5 mg strength tablets, blister-packed under aluminum foil in units of ten tablets and dosed on a daily basis. 10mg daily dosing throughout the trial.

Placebo

Placebo was given by continuous oral daily dosing of two 5 mg tablets.

Group Type PLACEBO_COMPARATOR

Everolimus Placebo

Intervention Type DRUG

Matching placebo was provided as a matching tablet and was also blister-packed under aluminum foil in units of ten.

Interventions

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Everolimus (RAD001)

Everolimus is used in 5 mg strength tablets, blister-packed under aluminum foil in units of ten tablets and dosed on a daily basis. 10mg daily dosing throughout the trial.

Intervention Type DRUG

Everolimus Placebo

Matching placebo was provided as a matching tablet and was also blister-packed under aluminum foil in units of ten.

Intervention Type DRUG

Other Intervention Names

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RAD001

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No