Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)

This study has been completed.
Sponsor:
Collaborator:
Novartis
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:
NCT00792766
First received: November 17, 2008
Last updated: September 17, 2013
Last verified: September 2013

November 17, 2008
September 17, 2013
December 2008
September 2013   (final data collection date for primary outcome measure)
RAD001 tolerance [ Time Frame: Every three months while on study drug ] [ Designated as safety issue: Yes ]
Same as current
Complete list of historical versions of study NCT00792766 on ClinicalTrials.gov Archive Site
Angiomyolipoma volume reduction [ Time Frame: Every year while on study drug ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)
Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

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 Lymphangioleiomyomatosis", CCHMC IRB #2008-0812 and who meet the criteria for this long-term follow-up study.

The hypothesis is that the drug will inhibit the growth of the angiomyolipomas and possibly even cause regression.

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 with LAM. They are fatty tumors that often occur in kidney or liver as well as other parts of the body. They can grow and cause damage to surrounding kidney tissue and even renal failure. They may also leak blood causing potentially life-threatening hemorrhage.

Laboratory studies have shown that RAD001 suppresses the chemical that cause tumors to grow in tuberous sclerosis and sporadic LAM. The use of RAD001 to treat angiomyolipomas in tuberous sclerosis or sporadic LAM is considered experimental.

Interventional
Phase 1
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Tuberous Sclerosis
  • Angiolipoma
Drug: everolimus (RAD001)
Subjects will resume the dosing regimen that they were receiving at the completion of the initial RAD001 study.
Other Name: RAD001
Experimental: RAD001
Intervention: Drug: everolimus (RAD001)
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
20
September 2013
September 2013   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • 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 Criteria:

  • 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.
Both
18 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00792766
CCHMC IRB# 2008-0333
Yes
Children's Hospital Medical Center, Cincinnati
Children's Hospital Medical Center, Cincinnati
Novartis
Principal Investigator: John Bissler, MD LeBonheur Children's Hospital
Children's Hospital Medical Center, Cincinnati
September 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP