NCT01217125

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.

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Oct 2008

Typical duration for phase_4

Status
completed

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, 2008

Completed
2 years until next milestone

First Submitted

Initial submission to the registry

October 6, 2010

Completed
2 days until next milestone

First Posted

Study publicly available on registry

October 8, 2010

Completed
7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2011

Completed
Last Updated

March 8, 2012

Status Verified

March 1, 2012

Enrollment Period

2.6 years

First QC Date

October 6, 2010

Last Update Submit

March 7, 2012

Conditions

Angiomyolipoma

Keywords

tuberousclerosis, angiomyolipoma, rapamycin

Outcome Measures

Primary Outcomes (1)

  • Angiomyolipoma volume

    Measured by MRI

    2 years

Secondary Outcomes (1)

  • skin lesions, AML complications,

    2 years

Interventions

SirolimusDRUG

plasma levels between 4 and 8

Also known as: Rapamune

Eligibility Criteria

Age10 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of tuberousclerosis Angiomyolipoma \>2cm age\>10 years

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (1)

  • 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.

    PMID: 23140536DERIVED

MeSH Terms

Conditions

Angiomyolipoma

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Neoplasms, Adipose TissueNeoplasms, Connective and Soft TissueNeoplasms by Histologic TypeNeoplasmsPerivascular Epithelioid Cell Neoplasms

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Study Officials

  • Roser Torra, MD,PhD

    Fundacio Puigvert, Barcelona

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD, PhD

Study Record Dates

First Submitted

October 6, 2010

First Posted

October 8, 2010

Study Start

October 1, 2008

Primary Completion

May 1, 2011

Study Completion

May 1, 2011

Last Updated

March 8, 2012

Record last verified: 2012-03