Rapamycin In Angiomyolipomas In Patients With Tuberous Sclerosis
Primary Purpose
Angiomyolipoma
Status
Completed
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
Sirolimus
Sponsored by
About this trial
This is an interventional treatment trial for Angiomyolipoma focused on measuring tuberousclerosis, angiomyolipoma, rapamycin
Eligibility Criteria
Inclusion Criteria:
Diagnosis of tuberousclerosis Angiomyolipoma >2cm age>10 years
Exclusion Criteria:
Creatinine >4 No recent AML bleeding No hepatic abnormalities (liver tests 2fold) Hematocrit<27% Thrombocytopenia (<100.000/mm3) Leukopenia (<3000/mm3) Ischemic cardiopathy Recent surgery (2 months prior to enrollment) Pregnancy Serum cholesterol over 7.8 mmol/l or hypertriglyceridemia fasten(>4.6 mmol/l) non controlled with drugs malignancy in the previous 2 years allergy to macrolides
Sites / Locations
Outcomes
Primary Outcome Measures
Angiomyolipoma volume
Measured by MRI
Secondary Outcome Measures
skin lesions, AML complications,
Evaluate skin lesions Collect event realted to AML
Full Information
NCT ID
NCT01217125
First Posted
October 6, 2010
Last Updated
March 7, 2012
Sponsor
Fundacio Puigvert
Collaborators
Ministry of Health, Spain
1. Study Identification
Unique Protocol Identification Number
NCT01217125
Brief Title
Rapamycin In Angiomyolipomas In Patients With Tuberous Sclerosis
Official Title
CLINICAL TRIAL TO DETERMINE THE EFFICACY AND SAFETY OF RAPAMYCIN IN ANGIOMYOLIPOMAS IN PATIENTS WITH TUBEROUS SCLEROSIS
Study Type
Interventional
2. Study Status
Record Verification Date
March 2012
Overall Recruitment Status
Completed
Study Start Date
October 2008 (undefined)
Primary Completion Date
May 2011 (Actual)
Study Completion Date
May 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Fundacio Puigvert
Collaborators
Ministry of Health, Spain
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to determine whether rapamycin is safe and effective in the treatment of renal angiomyolipomas in patients with tuberousclerosis.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Angiomyolipoma
Keywords
tuberousclerosis, angiomyolipoma, rapamycin
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
18 (Actual)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Sirolimus
Other Intervention Name(s)
Rapamune
Intervention Description
plasma levels between 4 and 8
Primary Outcome Measure Information:
Title
Angiomyolipoma volume
Description
Measured by MRI
Time Frame
2 years
Secondary Outcome Measure Information:
Title
skin lesions, AML complications,
Description
Evaluate skin lesions Collect event realted to AML
Time Frame
2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of tuberousclerosis Angiomyolipoma >2cm age>10 years
Exclusion Criteria:
Creatinine >4 No recent AML bleeding No hepatic abnormalities (liver tests 2fold) Hematocrit<27% Thrombocytopenia (<100.000/mm3) Leukopenia (<3000/mm3) Ischemic cardiopathy Recent surgery (2 months prior to enrollment) Pregnancy Serum cholesterol over 7.8 mmol/l or hypertriglyceridemia fasten(>4.6 mmol/l) non controlled with drugs malignancy in the previous 2 years allergy to macrolides
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Roser Torra, MD,PhD
Organizational Affiliation
Fundacio Puigvert, Barcelona
Official's Role
Principal Investigator
12. IPD Sharing Statement
Citations:
PubMed Identifier
23140536
Citation
Cabrera-Lopez C, Marti T, Catala V, Torres F, Mateu S, Ballarin J, Torra R. Assessing the effectiveness of rapamycin on angiomyolipoma in tuberous sclerosis: a two years trial. Orphanet J Rare Dis. 2012 Nov 11;7:87. doi: 10.1186/1750-1172-7-87.
Results Reference
derived
Learn more about this trial
Rapamycin In Angiomyolipomas In Patients With Tuberous Sclerosis
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