Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

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Brief Title

Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

Official Title

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

Brief Summary

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

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 Phase

Phase 2

Study Type

Interventional


Primary Outcome

Volume of Angiomyolipomata


Condition

Tuberous Sclerosis

Intervention

Rapamycin, sirolimus


Publications

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

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

25

Start Date

December 2002

Completion Date

March 2006


Eligibility Criteria

        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
      

Gender

All

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

John Bissler, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00457808

Organization ID

CCHMC IRB # 02-10-16

Secondary IDs

R21CA103486


Study Sponsor

Children's Hospital Medical Center, Cincinnati

Collaborators

 The LAM Foundation

Study Sponsor

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


Verification Date

April 2007