Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

Cincinnati Children's Hospital Medical Center

Status and phase: Completed; Phase 2

Conditions: Lymphangioleiomyomatosis; Tuberous Sclerosis

Treatments: Drug: Rapamycin, sirolimus

Study type: Interventional

Funder types: Other

Identifiers

NCT00457808

R21CA103486 (U.S. NIH Grant/Contract)

CCHMC IRB # 02-10-16

Details and patient eligibility

About

The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.

Full 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.

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)