Lymphangioleiomyomatosis, Tuberous Sclerosis
Conditions
Keywords
TSC and LAM, Tuberous Sclerosis Complex and sporadic LAM
Brief summary
The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.
Detailed 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.
Interventions
DRUGRapamycin, sirolimus
Sponsors
The LAM Foundation
Tuberous Sclerosis Alliance
Children's Hospital Medical Center, Cincinnati
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Volume of Angiomyolipomata | — |
Countries
United States
Outcome results
None listed