Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

Children's Hospital Medical Center, Cincinnati25 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Tuberous Sclerosis Lymphangioleiomyomatosis

Interventions

Rapamycin, sirolimusDRUG

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (1)

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, United States

Outcomes

Primary Outcomes

Volume of Angiomyolipomata

Data from ClinicalTrials.gov

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