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

This study has been completed.
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:
NCT00457964
First received: April 6, 2007
Last updated: September 23, 2013
Last verified: September 2013
  Purpose

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.


Condition Intervention Phase
Tuberous Sclerosis
Lymphangioleiomyomatosis
Drug: RAD001
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: RAD001 Therapy of Angiomyolipomata 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:
  • Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Enrollment: 36
Study Start Date: August 2005
Study Completion Date: July 2013
Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Administration of RAD001 Drug: RAD001
5 and 10 mg/day or 30, 50, 70mg/week

Detailed Description:

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

  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
  • 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00457964

Locations
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Novartis Pharmaceuticals
Investigators
Principal Investigator: John J. Bissler, M.D. LaBonheur Children's Hospital
  More Information

No publications provided

Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT00457964     History of Changes
Other Study ID Numbers: CCHMC IRB #2008-0812(04-07-22)
Study First Received: April 6, 2007
Last Updated: September 23, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Hospital Medical Center, Cincinnati:
Tuberous Sclerosis Complex (TSC)
Lymyphangioleiomyomatosis
Mammalian Target of Rapamycin (mTOR)
RAD001
Angiomyolipmas

Additional relevant MeSH terms:
Angiomyolipoma
Lymphangioleiomyomatosis
Sclerosis
Tuberous Sclerosis
Congenital Abnormalities
Genetic Diseases, Inborn
Hamartoma
Heredodegenerative Disorders, Nervous System
Immune System Diseases
Immunoproliferative Disorders
Lymphangiomyoma
Lymphatic Diseases
Lymphatic Vessel Tumors
Lymphoproliferative Disorders
Malformations of Cortical Development
Neoplasms
Neoplasms by Histologic Type
Neoplasms, Adipose Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Nervous System Diseases
Nervous System Malformations
Neurocutaneous Syndromes
Neurodegenerative Diseases
Pathologic Processes
Perivascular Epithelioid Cell Neoplasms
Everolimus
Sirolimus
Anti-Bacterial Agents

ClinicalTrials.gov processed this record on October 21, 2014