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

Tuberous Sclerosis Clinical Trial

Official title:

RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00457964
• Other study ID #: CCHMC IRB #2008-0812(04-07-22)
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: April 6, 2007
• Last updated: September 23, 2013
• Start date: August 2005
• Est. completion date: July 2013

Study information

• Verified date: September 2013
• Source: Children's Hospital Medical Center, Cincinnati
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 36
• Est. completion date: July 2013
• Est. primary completion date: July 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Angiomyolipoma
• Lymphangioleiomyomatosis
• Sclerosis
• Tuberous Sclerosis

Intervention

Drug:
• RAD001
5 and 10 mg/day or 30, 50, 70mg/week

Locations

Country	Name	City	State
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio

Sponsors (2)

Lead Sponsor	Collaborator
Children's Hospital Medical Center, Cincinnati	Novartis Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline		12 months	No