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A Phase II Multi-Center Study of Rapamycin for Treating Kidney Angiomyolipomas in TSC or LAM Patients


Phase 2
18 Years
65 Years
Open (Enrolling)
Both
Nonmalignant Neoplasm

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Trial Information

A Phase II Multi-Center Study of Rapamycin for Treating Kidney Angiomyolipomas in TSC or LAM Patients


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.

Inclusion 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

Type of Study:

Interventional

Study Design:

Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Objective response as assessed by RECIST criteria

Safety Issue:

No

Principal Investigator

Sandra Dabora, MD, PhD

Investigator Role:

Study Chair

Investigator Affiliation:

Dana-Farber/Brigham and Women's Cancer Center

Authority:

United States: Federal Government

Study ID:

CDR0000440080

NCT ID:

NCT00126672

Start Date:

June 2005

Completion Date:

Related Keywords:

  • Nonmalignant Neoplasm
  • angiomyolipoma
  • Neoplasms
  • Angiomyolipoma

Name

Location

Cincinnati Children's Hospital Medical CenterCincinnati, Ohio  45229-3039
University of Texas Southwestern Medical Center at DallasDallas, Texas  75235-8897
Massachusetts General HospitalBoston, Massachusetts  02114-2617
Connecticut Children's Medical CenterHartford, Connecticut  06106
New York University Medical CenterNew York, New York  10016
Tuberous Sclerosis Clinic at Loma Linda University Medical CenterLoma Linda, California  92350