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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00792766
• Date of registration: 17/11/2008
• Prospective Registration: Yes
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)
• Scientific title: Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
• Date of first enrolment: December 2008
• Target sample size: 20
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00792766
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: John Bissler, MD
• Affiliation: LeBonheur Children's Hospital

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: 65 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Angiolipoma

Tuberous Sclerosis

Intervention(s)

Drug: everolimus (RAD001)

Primary Outcome(s)

RAD001 tolerance [Time Frame: Every three months while on study drug]

Secondary Outcome(s)

Angiomyolipoma volume reduction [Time Frame: Every year while on study drug]

Secondary ID(s)

CCHMC IRB# 2008-0333

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Novartis