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Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

This study has been completed.
The LAM Foundation
Tuberous Sclerosis Alliance
Information provided by:
Children's Hospital Medical Center, Cincinnati Identifier:
First received: April 6, 2007
Last updated: NA
Last verified: April 2007
History: No changes posted
The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.

Condition Intervention Phase
Tuberous Sclerosis Lymphangioleiomyomatosis Drug: Rapamycin, sirolimus Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

Resource links provided by NLM:

Further study details as provided by Children's Hospital Medical Center, Cincinnati:

Primary Outcome Measures:
  • Volume of Angiomyolipomata

Estimated Enrollment: 25
Study Start Date: December 2002
Study Completion Date: March 2006
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.

Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
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Please refer to this study by its identifier: NCT00457808

United States, Ohio
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
The LAM Foundation
Tuberous Sclerosis Alliance
Principal Investigator: John Bissler, MD Cincinnati Childrens Hospital Medical Center
  More Information

Publications automatically indexed to this study by Identifier (NCT Number): Identifier: NCT00457808     History of Changes
Other Study ID Numbers: CCHMC IRB # 02-10-16
R21CA103486 ( U.S. NIH Grant/Contract )
Study First Received: April 6, 2007
Last Updated: April 6, 2007

Keywords provided by Children's Hospital Medical Center, Cincinnati:
Tuberous Sclerosis Complex and sporadic LAM

Additional relevant MeSH terms:
Tuberous Sclerosis
Pathologic Processes
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development, Group I
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Perivascular Epithelioid Cell Neoplasms
Neoplasms, Connective and Soft Tissue
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Anti-Bacterial Agents
Anti-Infective Agents processed this record on September 21, 2017