Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM
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The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.
Condition or disease
Drug: Rapamycin, sirolimus
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.
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Ages Eligible for Study:
18 Years to 65 Years (Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
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
At least one angiomyolipoma of 1 cm or greater in largest diameter
Use of continuous supplemental oxygen
Ongoing or planned pregnancy
Use of an investigational drug within the last 30 days of study entrance
Keywords provided by Children's Hospital Medical Center, Cincinnati:
TSC and LAM
Tuberous Sclerosis Complex and sporadic LAM
Additional relevant MeSH terms:
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Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development, Group I
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Perivascular Epithelioid Cell Neoplasms
Neoplasms, Connective and Soft Tissue
Immune System Diseases