Sirolimus in Treating Patients With Angiomyolipoma of the Kidney

The recruitment status of this study is unknown because the information has not been verified recently.
Verified October 2008 by National Cancer Institute (NCI).
Recruitment status was  Active, not recruiting
Sponsor:
Collaborator:
Information provided by:
National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:
NCT00126672
First received: August 2, 2005
Last updated: February 1, 2010
Last verified: October 2008
  Purpose

RATIONALE: Drugs used in chemotherapy, such as sirolimus, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This phase II trial is studying how well sirolimus works in treating patients with angiomyolipoma of the kidney.


Condition Intervention Phase
Nonmalignant Neoplasm
Drug: sirolimus
Phase 2

Study Type: Interventional
Study Design: Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II Multi-Center Study of Rapamycin for Treating Kidney Angiomyolipomas in TSC or LAM Patients

Resource links provided by NLM:


Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • Objective response as assessed by RECIST criteria [ Designated as safety issue: No ]
  • Toxicity as measured by NCI CTC [ Designated as safety issue: Yes ]

Estimated Enrollment: 36
Study Start Date: June 2005
Estimated Primary Completion Date: April 2010 (Final data collection date for primary outcome measure)
Detailed Description:

OBJECTIVES:

Primary

  • Determine the efficacy of sirolimus, in terms of objective response rate, in patients with angiomyolipoma of the kidney secondary to tuberous sclerosis (TSC) or lymphangioleiomyomatosis (LAM).
  • Determine the toxicity of this drug in these patients.

Secondary

  • Determine changes in other TSC lesions (e.g., tubers, subependymal giant cell astrocytomas, facial angiofibromas, or kidney cysts) in patients with TSC treated with this drug.
  • Determine changes in pulmonary disease in patients with LAM treated with this drug.

OUTLINE: This is a multicenter study.

Patients receive oral sirolimus once daily for 12 months in the absence of unacceptable toxicity.

After completion of study treatment, patients are followed at 6 months and 1 year.

PROJECTED ACCRUAL: A total of 13-35 patients will be accrued for this study within 3-30 months.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

DISEASE CHARACTERISTICS:

  • Diagnosis of angiomyolipoma of the kidney secondary to tuberous sclerosis (TSC) or lymphangioleiomyomatosis (LAM)*

    • Tumor ≥ 2 cm by MRI NOTE: *Diagnosis of LAM by chest CT scan
  • No angiomyolipoma-related bleeding or impending bleed
  • No evidence of severe LAM, defined as dependence on continuous oxygen
  • Untreated renal cell carcinoma

PATIENT CHARACTERISTICS:

Age

  • 18 to 65

Performance status

  • Not specified

Life expectancy

  • Not specified

Hematopoietic

  • Hematocrit > 27%
  • ANC > 1,500
  • Platelet count > 100,000

Hepatic

  • SGOT and SGPT < 2 times normal
  • Bilirubin < 2 times normal
  • Alkaline phosphatase < 2 times normal

Renal

  • eGFR 30 or higher
  • No evidence of accelerating renal dysfunction
  • No acute renal failure

Cardiovascular

  • No history of coronary artery disease

Pulmonary

  • See Disease Characteristics

Other

  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective barrier contraception
  • No unstable seizures, defined as a recent change in seizure pattern or change in antiepileptic drug regimen
  • No active infection

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • Not specified

Chemotherapy

  • Not specified

Endocrine therapy

  • Not specified

Radiotherapy

  • Not specified

Surgery

  • Not specified

Other

  • More than 30 days since prior investigational agents
  • More than 6 months since prior vascular embolization therapy for treatment of kidney angiomyolipomas
  • No concurrent chronic use of diltiazem, ketoconazole, or rifampin
  • No other concurrent investigational agents
  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: NCT00126672

Locations
United States, California
Tuberous Sclerosis Clinic at Loma Linda University Medical Center
Loma Linda, California, United States, 92350
United States, Connecticut
Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, New York
New York University Medical Center
New York, New York, United States, 10016
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039
United States, Texas
University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, United States, 75390
Sponsors and Collaborators
Dana-Farber Cancer Institute
Investigators
Study Chair: Sandra Dabora, MD, PhD Dana-Farber/Brigham and Women's Cancer Center
  More Information

Additional Information:
No publications provided by National Cancer Institute (NCI)

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Sandra Dabora, Dana-Farber/Brigham and Women's Cancer Center
ClinicalTrials.gov Identifier: NCT00126672     History of Changes
Other Study ID Numbers: CDR0000440080, DFCI-04298, WYETH-C-0468H1-101899
Study First Received: August 2, 2005
Last Updated: February 1, 2010
Health Authority: United States: Federal Government

Keywords provided by National Cancer Institute (NCI):
angiomyolipoma

Additional relevant MeSH terms:
Angiomyolipoma
Neoplasms
Neoplasms by Histologic Type
Neoplasms, Adipose Tissue
Neoplasms, Connective and Soft Tissue
Perivascular Epithelioid Cell Neoplasms
Everolimus
Sirolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents
Antineoplastic Agents
Immunologic Factors
Immunosuppressive Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on October 23, 2014