Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM
NCT ID: NCT00490789
Last Updated: 2008-04-30
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE2
14 participants
INTERVENTIONAL
2005-10-31
2009-09-30
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Interventions
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sirolimus
daily oral sirolimus with dosage individualised by trough blood levels
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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)
18 Years
65 Years
ALL
No
Sponsors
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University of Nottingham
OTHER
St Georges Hospital Medical School
UNKNOWN
Royal Sussex County Hospital
OTHER
The Tuberous Sclerosis Association
OTHER
Wyeth is now a wholly owned subsidiary of Pfizer
INDUSTRY
Cardiff University
OTHER
Responsible Party
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Cardiff University
Principal Investigators
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Julian R Sampson, DM
Role: PRINCIPAL_INVESTIGATOR
Cardiff Univeristy
Locations
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University Hospital of Wales
Cardiff, Wales, United Kingdom
Royal Sussex County Hospital
Brighton, , United Kingdom
City Hospital
Nottingham, , United Kingdom
Countries
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References
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Davies DM, de Vries PJ, Johnson SR, McCartney DL, Cox JA, Serra AL, Watson PC, Howe CJ, Doyle T, Pointon K, Cross JJ, Tattersfield AE, Kingswood JC, Sampson JR. Sirolimus therapy for angiomyolipoma in tuberous sclerosis and sporadic lymphangioleiomyomatosis: a phase 2 trial. Clin Cancer Res. 2011 Jun 15;17(12):4071-81. doi: 10.1158/1078-0432.CCR-11-0445. Epub 2011 Apr 27.
Other Identifiers
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TESSTAL
Identifier Type: -
Identifier Source: org_study_id