Status:
COMPLETED
RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS
Lead Sponsor:
Dana-Farber Cancer Institute
Collaborating Sponsors:
National Cancer Institute (NCI)
Wyeth is now a wholly owned subsidiary of Pfizer
Conditions:
Nonmalignant Neoplasm
Tuberous Sclerosis
Eligibility:
All Genders
3-65 years
Phase:
PHASE2
Brief Summary
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. Kidn...
Detailed Description
This research study is a Phase ll clinical trial. Phase II clinical trials test the effectiveness of an investigational drug to learn whether the drug works in treating a specific disease. "Investigat...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- 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.
- Exclusion criteria:
- 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.
Exclusion
Key Trial Info
Start Date :
December 20 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2010
Estimated Enrollment :
36 Patients enrolled
Trial Details
Trial ID
NCT00126672
Start Date
December 20 2005
End Date
April 1 2010
Last Update
April 25 2022
Active Locations (7)
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1
Loma Linda University School of Medicine
Loma Linda, California, United States, 92350
2
Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
3
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
4
New York University Medical Center
New York, New York, United States, 10016