Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

April 6, 2007 updated by: Children's Hospital Medical Center, Cincinnati

Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

The study design was an open label, phase I/II trial of sirolimus for one year followed by one year off therapy.Patients were seen at baseline,at two weeks to four weeks, and at 2,4,6,9,12,18 and 24 months. Angiomyolipomata imaging was performed at all but the two to four week visit. Complete pulmonary function tests and six-minute walk were obtained at baseline,6 or 9 month, 12 and 24 month visits, while simple spirometry only was performed at all other visits.

Study Type

Interventional

Enrollment

25

Phase

  • Phase 2

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, 45229-3039
        • Cincinnati Childrens 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
  • Between the gaes of 18 and 65 years
  • Competency to voluntarily consent
  • Clinically definite diagnosis of tuberous sclerosis or S-LAM
  • Adequate contraception
  • At least one angiomyolipoma of 1 cm or greater in largest diameter

Exclusion Criteria:

  • Use of continuous supplemental oxygen
  • Concurrent infection
  • Recent surgery
  • Ongoing or planned pregnancy
  • Lactation
  • Use of an investigational drug within the last 30 days of study entrance

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: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Volume of Angiomyolipomata

Collaborators and Investigators

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

Sponsor

Children's Hospital Medical Center, Cincinnati

Collaborators

The LAM Foundation
Tuberous Sclerosis Alliance

Investigators

  • Principal Investigator: John Bissler, MD, Cincinnati Childrens Hospital Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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

December 1, 2002

Study Completion (Actual)

March 1, 2006

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)

April 9, 2007

Last Update Submitted That Met QC Criteria

April 6, 2007

Last Verified

April 1, 2007

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CCHMC IRB # 02-10-16
  • R21CA103486 (U.S. NIH Grant/Contract)

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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