RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS

Completed · Phase 2

Conditions studied: Nonmalignant Neoplasm, Tuberous Sclerosis, Lymphangioleimyomatosis, Kidney Angiomyolipoma

In brief

This research study is evaluating a drug called rapamycin as a possible treatment for the lumps (or tumors) that form in the kidneys, called angiomyolipomas, in people who have either TSC or LAM. Kidney angiomyolipomas are tumors that are made up of blood vessels, muscle and fat. Rapamycin has been approved to treat other diseases, but it is investigational for treating kidney angiomyolipomas. Investigational means that it is being as a possible treatment for kidney angiomyolipomas but is not currently approved by the U.S. Food and Drug Administration (FDA) for treating this disease.

Key facts

Study ID

NCT00126672

Run by

Dana-Farber Cancer Institute

People needed

36

Starts

2005-12-20

Expected to finish

2010-04-01

Last updated by the study team

2022-04-25

Who can join

Age: 3 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age 3-65 years old (females of reproductive age must not be pregnant or breastfeeding)
• Kidney ANGIOMYOLIPOMA 2 cm or greater on baseline MRI (a CT scan may be used for patients who cannot undergo MRI imaging)
• No evidence of severe LAM (not on continuous oxygen)
• Informed consent, including consent for submission of blood, urine and tissue samples as described in the appendix.
• Adequate renal and liver function (eGFR of 30 or higher, SGOT, SGPT, TBili, Alk Phos all<2x normal)
• HCT>27%
• ANC > 1500 and platelet count >100,000
• Diagnosis of TSC or LAM (diagnosis of TSC using revised diagnostic criteria [45], diagnosis of LAM made by chest CT scan and reviewed by a pulmonologist).
• Fertility/Reproductive issues: The effects of rapamycin on the developing fetus at the doses used in this study are unknown. For this reason, rapamycin should not be taken during pregnancy. Participants who are fertile must maintain adequate contraception while they are taking rapamycin and for twelve weeks after stopping the drug. Acceptable contraceptive measures include prior hysterectomy, oophorectomy or tubal ligation, complete abstinence, barrier methods which include both a cervical diaphragm and spermicidal jelly, and progestin based contraceptives. Pregnancy tests will be obtained at enrollment and during study visits at 8 weeks, 16 weeks, 24 weeks, 32 weeks, 40 weeks, and 52 weeks.
• Note: Eligibility requirement of ECOG PS of 0 or 1 has been removed to allow participation of those subjects with TSC who are classified as ECOG PS of 2,3, or 4 because of cognitive impairment rather than progressive disease. This change allows site Pl's to individualize decision making on whether or not to enroll such subjects after discussing details of the study with prospective participant and legal guardian. The decision to enroll individuals with ECOG PS of 2,3, or 4 will be at the discretion of the Principal Investigator.

You may not qualify if…

• Unstable seizures (defined as changes in anti-epileptics OR increase in frequency and/or severity or seizures in the 60 days prior to study entry)
• Significant bleed associated with kidney angiomyolipoma(s) (defined as bleed associated with shock OR requiring a blood transfusion in the 30 days prior to study entry)
• Severe LAM (defined as dependent on continuous supplemental oxygen)
• Evidence for accelerating renal dysfunction or acute renal failure
• Diagnosis of Renal Cell Cancer that has not been treated (additional clarification: individuals with a prior history of renal cancer who have had appropriate surgery and have no evidence of metastatic disease can be enrolled)
• Active infection
• Patients will be excluded if they have been treated with any investigational agent in the 30 days prior to study entry
• Patients may not be treated with other investigational agents while on study
• Prior history of coronary artery disease
• Vascular embolization for treatment of kidney angiomyolipoma(s) within 6 months
• Patients who must take diltiazem, ketoconazole or rifampin chronically will be excluded because of known drug interactions. Both diltiazem and ketoconazole are strong inhibitors of CYP3A4 and are known to increase rapamycin levels. Rifampin is a known CYP3A4 and P-glycoprotein inducer and is known to significantly reduce rapamycin levels.

Where it is running

• Loma Linda University School of Medicine — Loma Linda, California, United States
• Connecticut Children's Medical Center — Hartford, Connecticut, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• New York University Medical Center — New York, New York, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• University of Pennsylvania Medical Center — Philadelphia, Pennsylvania, United States
• University of Texas Southwestern Medical Center at Dallas — Dallas, Texas, United States

Full record on ClinicalTrials.gov

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