Rapamycin In Angiomyolipomas In Patients With Tuberous Sclerosis

This study has been completed.
Sponsor:
Collaborator:
Ministry of Health, Spain
Information provided by (Responsible Party):
Roser Torra, Fundacio Puigvert
ClinicalTrials.gov Identifier:
NCT01217125
First received: October 6, 2010
Last updated: March 7, 2012
Last verified: March 2012
  Purpose

The purpose of this study is to determine whether rapamycin is safe and effective in the treatment of renal angiomyolipomas in patients with tuberousclerosis.


Condition Intervention Phase
Angiomyolipoma
Drug: Sirolimus
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: CLINICAL TRIAL TO DETERMINE THE EFFICACY AND SAFETY OF RAPAMYCIN IN ANGIOMYOLIPOMAS IN PATIENTS WITH TUBEROUS SCLEROSIS

Resource links provided by NLM:


Further study details as provided by Fundacio Puigvert:

Primary Outcome Measures:
  • Angiomyolipoma volume [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Measured by MRI


Secondary Outcome Measures:
  • skin lesions, AML complications, [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Evaluate skin lesions Collect event realted to AML


Enrollment: 18
Study Start Date: October 2008
Study Completion Date: May 2011
Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Sirolimus
    plasma levels between 4 and 8
    Other Name: Rapamune
  Eligibility

Ages Eligible for Study:   10 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01217125

Sponsors and Collaborators
Fundacio Puigvert
Ministry of Health, Spain
Investigators
Principal Investigator: Roser Torra, MD,PhD Fundacio Puigvert, Barcelona
  More Information

No publications provided

Responsible Party: Roser Torra, MD, PhD, Fundacio Puigvert
ClinicalTrials.gov Identifier: NCT01217125     History of Changes
Other Study ID Numbers: EudraCT 2007-005978-30
Study First Received: October 6, 2010
Last Updated: March 7, 2012
Health Authority: Spain: Spanish agency of medicine and health care products

Keywords provided by Fundacio Puigvert:
tuberousclerosis, angiomyolipoma, rapamycin

Additional relevant MeSH terms:
Sclerosis
Tuberous Sclerosis
Angiomyolipoma
Pathologic Processes
Hamartoma
Neoplasms
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Neoplasms, Adipose Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Perivascular Epithelioid Cell Neoplasms
Sirolimus
Everolimus
Antibiotics, Antineoplastic
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Antifungal Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents

ClinicalTrials.gov processed this record on July 29, 2014