Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

Tuberous Sclerosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00457808
• Other study ID #: CCHMC IRB # 02-10-16
• Secondary ID: R21CA103486
• Status: Completed
• Phase: Phase 2
• First received: April 6, 2007
• Last updated: April 6, 2007
• Start date: December 2002
• Est. completion date: March 2006

Study information

• Verified date: April 2007
• 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 study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: March 2006
• 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

• 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

Study Design

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

Related Conditions & MeSH terms

• Lymphangioleiomyomatosis
• Sclerosis
• Tuberous Sclerosis

Intervention

Drug:
• Rapamycin, sirolimus

Locations

Country	Name	City	State
United States	Cincinnati Childrens Hospital Medical Center	Cincinnati	Ohio

Sponsors (3)

Lead Sponsor	Collaborator
Children's Hospital Medical Center, Cincinnati	The LAM Foundation, Tuberous Sclerosis Alliance

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Volume of Angiomyolipomata