Rapamycin In Angiomyolipomas In Patients With Tuberous Sc... | NCT01217125

Studies
Rapamycin In Angiomyolipomas In Patients With Tuberous Sclerosis

NCT01217125

Sponsor

Fundacio Puigvert

Status

Completed

Last Update Posted

Mar 8, 2012Estimated

Enrollment

18Actual

Phase

Phase 4

Conditions

Angiomyolipoma

Interventions

Sirolimus

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2012

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Oct 2008

Primary Completion Date

May 2011Actual

Completion Date

May 2011Actual

First Submitted Date

Oct 6, 2010

First Submission Date that Met QC Criteria

Oct 7, 2010

First Posted Date

Oct 8, 2010Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 7, 2012

Last Update Posted Date

Mar 8, 2012Estimated

Name	Class
Ministry of Health, Spain	OTHER_GOV

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 4

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

18Actual

Name	Type	Description	Arm Group Labels	Other Names
Sirolimus	Drug	plasma levels between 4 and 8		Rapamune

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Angiomyolipoma volume	Measured by MRI	2 years

Secondary Outcomes

Measure	Description	Time Frame
skin lesions, AML complications,	Evaluate skin lesions Collect event realted to AML	2 years

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

10 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Roser Torra, MD,PhD	Fundacio Puigvert, Barcelona	Principal Investigator

Locations

Not provided

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
23140536	Derived	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.

ID	Term
D018205	Neoplasms, Adipose Tissue
D018204	Neoplasms, Connective and Soft Tissue
D009370	Neoplasms by Histologic Type
D009369	Neoplasms

ID	Term
D018942	Macrolides
D007783	Lactones
D009930	Organic Chemicals