- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00490789
Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM (TESSTAL)
April 29, 2008 updated by: Cardiff University
A Trial of the Efficacy and Safety of Sirolimus(Rapamycin)Therapy for Renal Angiomyolipmoas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
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.
Study Overview
Status
Unknown
Conditions
Intervention / Treatment
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
Study Type
Interventional
Enrollment (Anticipated)
14
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Brighton, United Kingdom, BN2 5BE
- Royal Sussex County Hospital
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Nottingham, United Kingdom, NG5 1PB
- City Hospital
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Wales
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Cardiff, Wales, United Kingdom, CF14 4XN
- University Hospital of Wales
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 65 years (ADULT, OLDER_ADULT)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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)
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NON_RANDOMIZED
- Interventional Model: SINGLE_GROUP
- Masking: NONE
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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
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assessments at baseline and 2,6,12 and 24 months
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toxicity graded by National Cancer Institute's Common Terminology Criteria for Adverse Events
Time Frame: throughout study
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throughout study
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
respiratory function tests (FEV1, FVC, DLCO), cognitive function (memory, executive skills)
Time Frame: 2 years
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2 years
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Julian R Sampson, DM, Cardiff Univeristy
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
October 1, 2005
Primary Completion (ANTICIPATED)
September 1, 2009
Study Completion (ANTICIPATED)
September 1, 2009
Study Registration Dates
First Submitted
June 21, 2007
First Submitted That Met QC Criteria
June 21, 2007
First Posted (ESTIMATE)
June 25, 2007
Study Record Updates
Last Update Posted (ESTIMATE)
April 30, 2008
Last Update Submitted That Met QC Criteria
April 29, 2008
Last Verified
April 1, 2008
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Immune System Diseases
- Neoplasms, Connective and Soft Tissue
- Neoplasms by Histologic Type
- Neoplasms
- Lymphoproliferative Disorders
- Lymphatic Diseases
- Immunoproliferative Disorders
- Congenital Abnormalities
- Genetic Diseases, Inborn
- Neurodegenerative Diseases
- Heredodegenerative Disorders, Nervous System
- Neoplastic Syndromes, Hereditary
- Malformations of Cortical Development, Group I
- Malformations of Cortical Development
- Nervous System Malformations
- Lymphangiomyoma
- Lymphatic Vessel Tumors
- Perivascular Epithelioid Cell Neoplasms
- Neurocutaneous Syndromes
- Hamartoma
- Neoplasms, Multiple Primary
- Sclerosis
- Lymphangioleiomyomatosis
- Tuberous Sclerosis
- Physiological Effects of Drugs
- Anti-Infective Agents
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Anti-Bacterial Agents
- Antibiotics, Antineoplastic
- Antifungal Agents
- Sirolimus
Other Study ID Numbers
- TESSTAL
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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