Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM
TESSTAL
A Trial of the Efficacy and Safety of Sirolimus(Rapamycin)Therapy for Renal Angiomyolipmoas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
1 other identifier
interventional
14
1 country
3
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Oct 2005
Typical duration for phase_2
3 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 1, 2005
CompletedFirst Submitted
Initial submission to the registry
June 21, 2007
CompletedFirst Posted
Study publicly available on registry
June 25, 2007
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2009
CompletedStudy Completion
Last participant's last visit for all outcomes
September 1, 2009
CompletedApril 30, 2008
April 1, 2008
3.9 years
June 21, 2007
April 29, 2008
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
longest diameter of renal angiomyolipomas assessed by MRI scan, toxicity graded by National Cancer Institute's Common Terminology Criteria for Adverse Events v3.0
assessments at baseline and 2,6,12 and 24 months
toxicity graded by National Cancer Institute's Common Terminology Criteria for Adverse Events
throughout study
Secondary Outcomes (1)
respiratory function tests (FEV1, FVC, DLCO), cognitive function (memory, executive skills)
2 years
Interventions
daily oral sirolimus with dosage individualised by trough blood levels
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
- +3 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Cardiff Universitylead
- University of Nottinghamcollaborator
- St Georges Hospital Medical Schoolcollaborator
- Royal Sussex County Hospitalcollaborator
- The Tuberous Sclerosis Associationcollaborator
- Wyeth is now a wholly owned subsidiary of Pfizercollaborator
Study Sites (3)
University Hospital of Wales
Cardiff, Wales, CF14 4XN, United Kingdom
Royal Sussex County Hospital
Brighton, BN2 5BE, United Kingdom
City Hospital
Nottingham, NG5 1PB, United Kingdom
Related Publications (1)
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.
PMID: 21525172DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Julian R Sampson, DM
Cardiff Univeristy
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
Study Record Dates
First Submitted
June 21, 2007
First Posted
June 25, 2007
Study Start
October 1, 2005
Primary Completion
September 1, 2009
Study Completion
September 1, 2009
Last Updated
April 30, 2008
Record last verified: 2008-04