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Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

Phase 1/Phase 2
18 Years
65 Years
Open (Enrolling)
Tuberous Sclerosis, Angiolipoma

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Trial Information

Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

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

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.

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.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

RAD001 tolerance

Outcome Time Frame:

Every three months while on study drug

Safety Issue:


Principal Investigator

John Bissler, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Children's Hospital Medical Center, Cincinnati


United States: Food and Drug Administration

Study ID:

CCHMC IRB# 2008-0333



Start Date:

December 2008

Completion Date:

December 2013

Related Keywords:

  • Tuberous Sclerosis
  • Angiolipoma
  • Tuberous Sclerosis Complex (TSC)
  • Angiolipoma
  • mTOR
  • Everolimus
  • Sclerosis
  • Tuberous Sclerosis
  • Angiolipoma
  • Angiomyolipoma
  • Lymphangioleiomyomatosis



Cincinnati Children's Hospital Medical Center Cincinnati, Ohio  45229-3039