Status:
COMPLETED
Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati
Collaborating Sponsors:
Novartis
Conditions:
Tuberous Sclerosis
Angiolipoma
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
PHASE2
Brief Summary
This is an open label long term follow up study, open to those subjects who were previously enrolled in"RAD001 Therapy of Angiomyolipomata in Patients with Tuberous Sclerosis Complex and Sporadic Lymp...
Detailed Description
Tuberous sclerosis complex is a genetic disorder with a birth incidence of approximately one in six thousand. Angiomyolipomas occur in eighty percent of persons with TS, and in up to 60% of persons wi...
Eligibility Criteria
Inclusion
- Subjects must have met all eligibility criteria for the initial RAD001 protocol (CCHMC IRB 2008-0812)
- Subjects with documented angiomyolipoma volume decrease from baseline measures at the end of 12 months on study drug of thirty percent or more during the initial RAD001 protocol OR subjects with less than thirty percent decrease in angiomyolipomas at the end of 12 months on study drug but with documented improvement, or stabilization, of baseline clinical status per physical, pulmonary function and/or laboratory examination at the end of 12 months on study drug that was not maintained during a period of 12 or more months off study drug.
- If female and of child-bearing potential, documentation of negative pregnancy test prior to start of study drug
- Creatinine \<3 mg/dl, within 30 days prior to start of drug
Exclusion
- Inability to complete the initial RAD001 protocol (CCHMC IRB # 2008-0812) due to toxicities requiring discontinuation of treatment.
- Demonstrated an increase in the size of the angiomyolipoma from baseline at the end of 12 months on study drug on the initial RAD001 study.
- Significant hematologic or hepatic abnormality (i.e. ALT and AST \>2.5x ULN), serum albumin \<3 g/dl, HCT \<30%, platelets \<75,000/cumm, adjusted absolute neutrophil count \<1,000/cumm, total WBC \<3,000/cumm).
- Continuous requirement for supplemental oxygen.
- Intercurrent infection at initiation of RAD001.
- Embolization of angiomyolipoma within one month; any other recent surgery (within 2 months of initiation of RAD001).
- Pregnant or lactating women or women who plan on becoming pregnant during the course of this study due to unknown effects of RAD001 on the fetus.
- Inadequate contraception (participants who are fertile must maintain adequate contraception throughout the trial and for three months after stopping the drug). Acceptable contraceptive measures include non estrogen-containing birth control contraceptive regimen (progestin based contraceptives), prior hysterectomy, tubal ligation, complete abstinence, barrier methods which include a cervical diaphragm and spermicidal jelly, IUD, or vasectomy in partner.
- Use of an investigational drug, including rapamycin, within the last 30 days.
Key Trial Info
Start Date :
December 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2013
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT00792766
Start Date
December 1 2008
End Date
September 1 2013
Last Update
September 18 2013
Active Locations (1)
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1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229