RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM

September 23, 2013 updated by: Children's Hospital Medical Center, Cincinnati

RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

The purpose of this research study is to find out what effects RAD001 has on angiomyolipomas of a person with Tuberous Sclerosis Complex and to determine the safe dose of RAD001 without toxicity.

The hypothesis is that the drug will inhibit the growth of the angiomyolipomas and possibly even cause regression.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Tuberous sclerosis complex is a genetic disorder with a birth incidence of approximately one in six thousand. Angiomyolipomas occur in eighty percent of persons with TS, and in up to 60% of persons with LAM. They are fatty tumors that often occur in kidney or liver as well as other parts of the body. They can grow and cause damage to surrounding kidney tissue and even renal failure. They may also leak blood causing potentially life-threatening hemorrhage.

Laboratory studies have shown that RAD001 suppresses the chemical that cause tumors to grow in tuberous sclerosis and sporadic LAM.

The primary goal of this study is to evaluate the clinical effectiveness of RAD001 in a 24 month trial. Although the primary goal is to determine if the drug RAD001 has effects on angiomyolipomas, other diseases associated with tuberous sclerosis will be monitored too, specifically any change in involvement of your brain or lungs with tuberous sclerosis. The use of RAD001 to treat angiomyolipomas in tuberous sclerosis or sporadic LAM is considered experimental

Study Type

Interventional

Enrollment (Actual)

36

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Diagnosed with angiomyolipomas and have either Tuberous Sclerosis Complex or lymphangioleiomyomatosis
  • Angiomyolipoma one centimeter or greater in largest diameter
  • Between the ages of 18 and 65 years old.
  • If female, documentation of negative pregnancy test prior to enrollment and, where applicable, use of appropriate non-estrogen containing birth control contraceptive regimen while on study.
  • Adequate renal function (creatinine < 3 mg/dl)

Exclusion Criteria:

  • Pregnant or lactating women
  • Continuous requirement for supplemental oxygen
  • Surgery within past 2 months
  • Use of an investigational drug within last 30 days.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Administration of RAD001
Drug: RAD001
5 and 10 mg/day or 30, 50, 70mg/week

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline
Time Frame: 12 months
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's Hospital Medical Center, Cincinnati

Collaborators

Novartis Pharmaceuticals

Investigators

  • Principal Investigator: John J. Bissler, M.D., LaBonheur Children's Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2005

Primary Completion (ACTUAL)

July 1, 2013

Study Completion (ACTUAL)

July 1, 2013

Study Registration Dates

First Submitted

April 6, 2007

First Submitted That Met QC Criteria

April 6, 2007

First Posted (ESTIMATE)

April 9, 2007

Study Record Updates

Last Update Posted (ESTIMATE)

September 25, 2013

Last Update Submitted That Met QC Criteria

September 23, 2013

Last Verified

September 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CCHMC IRB #2008-0812(04-07-22)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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