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Sponsors and Collaborators: |
Cardiff University University of Nottingham St Georges Hospital Medical School Royal Sussex County Hospital Wales Gene Park The Tuberous Sclerosis Association Wyeth |
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Information provided by: | Cardiff University |
ClinicalTrials.gov Identifier: | NCT00490789 |
The purpose of this study is to determine the safety and efficacy of the mTOR inhibitor sirolimus as a treatment for renal angiomyolipomas in patients with tyberous sclerosis complex or sporadic lymphangioleiomyomatosis.
Condition | Intervention | Phase |
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Tuberous Sclerosis Lymphangioleiomyomatosis |
Drug: sirolimus |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | A Trial of the Efficacy and Safety of Sirolimus(Rapamycin)Therapy for Renal Angiomyolipmoas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis |
Estimated Enrollment: | 14 |
Study Start Date: | October 2005 |
Estimated Study Completion Date: | September 2009 |
Estimated Primary Completion Date: | September 2009 (Final data collection date for primary outcome measure) |
Inherited mutations of the TSC1 or TSC2 gene cause tuberous sclerosis while acquired (somatic) mutations of either gene are associated with sporadic lymphangioleiomyomatosis (LAM). Renal angiomyolipomas are a feature of both disorders. TSC1 and TSC2 regulate signalling through the mammalian target of rapamycin (mTOR) pathway. Inhibition of mTOR may result in a decrease in size of TSC 1/2 assciated lesions. We are treating patients with tuberous sclerosis or sporadic LAM with the mTOR inhibitor rapamycin in a non-randomised, open label pilot study of safety and efficacy. Change in size of renal angiomyolipomas is the primary end point
Ages Eligible for Study: | 18 Years to 65 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United Kingdom | |
City Hospital | |
Nottingham, United Kingdom, NG5 1PB | |
Royal Sussex County Hospital | |
Brighton, United Kingdom, BN2 5BE | |
United Kingdom, Wales | |
University Hospital of Wales | |
Cardiff, Wales, United Kingdom, CF14 4XN |
Principal Investigator: | Julian R Sampson, DM | Cardiff Univeristy |
Responsible Party: | Cardiff University ( Julian R Sampson ) |
Study ID Numbers: | TESSTAL |
Study First Received: | June 21, 2007 |
Last Updated: | April 29, 2008 |
ClinicalTrials.gov Identifier: | NCT00490789 |
Health Authority: | United Kingdom: Research Ethics Committee; United Kingdom: Medicines and Healthcare Products Regulatory Agency |
tuberous sclerosis lymphangioleiomyomatosis sirolimus |
angiomyolioma rapamycin mTOR |
Sirolimus Immunoproliferative Disorders Clotrimazole Nervous System Malformations Miconazole Tioconazole Lymphangiomyoma Sclerosis Neurodegenerative Diseases Bourneville syndrome |
Lymphangioleiomyomatosis Lymphatic Diseases Tuberous Sclerosis Heredodegenerative Disorders, Nervous System Genetic Diseases, Inborn Tuberous sclerosis Malformations of Cortical Development Congenital Abnormalities Lymphoproliferative Disorders Neurocutaneous Syndromes |
Anti-Infective Agents Neoplasms by Histologic Type Immunologic Factors Immune System Diseases Antineoplastic Agents Physiological Effects of Drugs Nervous System Diseases Antibiotics, Antineoplastic Immunosuppressive Agents |
Hamartoma Pharmacologic Actions Lymphatic Vessel Tumors Anti-Bacterial Agents Neoplasms Pathologic Processes Therapeutic Uses Antifungal Agents |