MedDataX Logo

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

NCT ID: NCT00457964

Last Updated: 2013-09-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

36 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-08-31

Study Completion Date

2013-07-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Tuberous Sclerosis Lymphangioleiomyomatosis

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Tuberous Sclerosis Complex (TSC) Lymyphangioleiomyomatosis Mammalian Target of Rapamycin (mTOR) RAD001 Angiomyolipmas

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Administration of RAD001

Group Type EXPERIMENTAL

RAD001

Intervention Type DRUG

5 and 10 mg/day or 30, 50, 70mg/week

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

RAD001

5 and 10 mg/day or 30, 50, 70mg/week

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role collaborator

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

John J. Bissler, M.D.

Role: PRINCIPAL_INVESTIGATOR

LaBonheur Children's Hospital

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

CCHMC IRB #2008-0812(04-07-22)

Identifier Type: -

Identifier Source: org_study_id