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Trial record 4 of 30 for:    Tuberous Sclerosis
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Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

This study has been completed.
Sponsor:
Collaborators:
The LAM Foundation
Tuberous Sclerosis Alliance
Information provided by:
Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:
NCT00457808
First received: April 6, 2007
Last updated: NA
Last verified: April 2007
History: No changes posted
  Purpose

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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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:

U.S. FDA Resources

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.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00457808

Locations
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
Investigators
Principal Investigator: John Bissler, MD Cincinnati Childrens Hospital Medical Center
  More Information

No publications provided by Children's Hospital Medical Center, Cincinnati

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

ClinicalTrials.gov Identifier: NCT00457808     History of Changes
Other Study ID Numbers: CCHMC IRB # 02-10-16, R21CA103486
Study First Received: April 6, 2007
Last Updated: April 6, 2007
Health Authority: United States: Food and Drug Administration

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

Additional relevant MeSH terms:
Sclerosis
Tuberous Sclerosis
Lymphangioleiomyomatosis
Pathologic Processes
Hamartoma
Neoplasms
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Lymphangiomyoma
 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
Sirolimus
Everolimus
Antibiotics, Antineoplastic
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Antifungal Agents

ClinicalTrials.gov processed this record on May 19, 2013