Sirolimus in Treating Patients With Angiomyolipoma of the Kidney - Full Text View

Sponsor:	Dana-Farber Cancer Institute
Collaborator:	National Cancer Institute (NCI)
Information provided by:	National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:	NCT00126672

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
Precancerous/Nonmalignant Condition	Drug: sirolimus	Phase II

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

Resource links provided by NLM:

MedlinePlus related topics: Cancer

Drug Information available for: Sirolimus

U.S. FDA Resources

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

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

National Cancer Institute (NCI)

Investigators

Study Chair:

Sandra Dabora, MD, PhD

Dana-Farber/Brigham and Women's Cancer Center

More Information

Additional Information:

Clinical trial summary from the National Cancer Institute's PDQ® database This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Dana-Farber/Brigham and Women's Cancer Center ( Sandra Dabora )
Study ID Numbers:	CDR0000440080, DFCI-04298, WYETH-C-0468H1-101899
Study First Received:	August 2, 2005
Last Updated:	November 1, 2008
ClinicalTrials.gov Identifier:	NCT00126672 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by National Cancer Institute (NCI):

angiomyolipoma

Additional relevant MeSH terms:

Sirolimus
Anti-Infective Agents
Neoplasms by Histologic Type
Immunologic Factors
Precancerous Conditions
Antineoplastic Agents
Physiological Effects of Drugs
Angiomyolipoma
Antibiotics, Antineoplastic

Immunosuppressive Agents
Pharmacologic Actions
Anti-Bacterial Agents
Neoplasms, Connective and Soft Tissue
Neoplasms
Therapeutic Uses
Antifungal Agents
Neoplasms, Adipose Tissue

ClinicalTrials.gov processed this record on November 30, 2009