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Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM (TESSTAL)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00490789
Recruitment Status : Unknown
Verified April 2008 by Cardiff University.
Recruitment status was:  Active, not recruiting
First Posted : June 25, 2007
Last Update Posted : April 30, 2008
University of Nottingham
St Georges Hospital Medical School
Royal Sussex County Hospital
The Tuberous Sclerosis Association
Wyeth is now a wholly owned subsidiary of Pfizer
Information provided by:
Cardiff University

Brief Summary:
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 or disease Intervention/treatment Phase
Tuberous Sclerosis Lymphangioleiomyomatosis Drug: sirolimus Phase 2

Detailed Description:
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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 14 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : October 2005
Estimated Primary Completion Date : September 2009
Estimated Study Completion Date : September 2009

Intervention Details:
  • Drug: sirolimus
    daily oral sirolimus with dosage individualised by trough blood levels
    Other Names:
    • rapamune
    • rapamycin

Primary Outcome Measures :
  1. longest diameter of renal angiomyolipomas assessed by MRI scan, toxicity graded by National Cancer Institute's Common Terminology Criteria for Adverse Events v3.0 [ Time Frame: assessments at baseline and 2,6,12 and 24 months ]
  2. toxicity graded by National Cancer Institute's Common Terminology Criteria for Adverse Events [ Time Frame: throughout study ]

Secondary Outcome Measures :
  1. respiratory function tests (FEV1, FVC, DLCO), cognitive function (memory, executive skills) [ Time Frame: 2 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • If female, documentation of negative pregnancy test prior to enrolment.
  • Participants, including males, must use an effective form of contraception, whilst taking sirolimus and for twelve weeks after stopping the drug
  • One or more renal angiomyolipomata of at least two centimetres or greater in largest diameter
  • Adequate renal function :glomerular filtration rate > 40 ml/min
  • Clinically definite diagnosis of tuberous sclerosis (modified Gomez criteria) or sporadic LAM (biopsy-proven or compatible high resolution chest CT scan and respiratory function tests.)
  • Signed and dated informed consent

Exclusion Criteria:

  • History of non-compliance or inability to give informed consent
  • Significant haematological or hepatic abnormality (i.e. transaminase levels > 150 i.u./L serum albumin < 30 g/L, haematocrit< 30%, platelets < 100,000/ mm3, adjusted absolute neutrophil count < 1,500/mm3, total WBC < 3,000/ mm3)
  • Greater than 1 g proteinuria daily
  • Multiple bilateral AMLs, where individual lesions cannot be distinguished
  • Renal haemorrhage within preceding year
  • In those who have had a renal haemorrhage, known conservatively managed renal aneurysm(s) greater than 10mm
  • Patients who have had embolisation for AML(s) within the preceding 6 months
  • Patients who are unable to walk 100 metres on the flat
  • Continuous requirement for supplemental oxygen
  • Patients who have had or are being considered for organ transplant
  • Uncontrolled hyperlipidaemia
  • Intercurrent infection at initiation of Sirolimus
  • Surgery within last 2 months
  • Pregnant or lactating women
  • Use of an investigational drug within the last 30 days
  • Change in anti epileptic drug medication within the last 3 months
  • Likely to need vaccination e.g. for travel during the course of the trial (except for influenza vaccine in patients with LAM)
  • Current usage of strong inhibitors of CYP3AE ( such as ketoconazole, voriconazole, itraconazole, tilithromycin or clarithromycin) or strong inducers (such as rifampicin or rifabutin)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00490789

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United Kingdom
University Hospital of Wales
Cardiff, Wales, United Kingdom, CF14 4XN
Royal Sussex County Hospital
Brighton, United Kingdom, BN2 5BE
City Hospital
Nottingham, United Kingdom, NG5 1PB
Sponsors and Collaborators
Cardiff University
University of Nottingham
St Georges Hospital Medical School
Royal Sussex County Hospital
The Tuberous Sclerosis Association
Wyeth is now a wholly owned subsidiary of Pfizer
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Principal Investigator: Julian R Sampson, DM Cardiff Univeristy
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Julian R Sampson, Cardiff University Identifier: NCT00490789    
Other Study ID Numbers: TESSTAL
First Posted: June 25, 2007    Key Record Dates
Last Update Posted: April 30, 2008
Last Verified: April 2008
Keywords provided by Cardiff University:
tuberous sclerosis
Additional relevant MeSH terms:
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Tuberous Sclerosis
Pathologic Processes
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development, Group I
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Perivascular Epithelioid Cell Neoplasms
Neoplasms, Connective and Soft Tissue
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic