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

NCT00792766 | Completed Phase 1 DMC

• 1 other identifier: CCHMC IRB# 2008-0333
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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 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.

Conditions

Tuberous Sclerosis; Angiolipoma

Keywords

Tuberous Sclerosis Complex (TSC); Angiolipoma; mTOR; Everolimus

Outcome Measures

Primary Outcomes (1)

• RAD001 tolerance

  Every three months while on study drug

Secondary Outcomes (1)

• Angiomyolipoma volume reduction

  Every year while on study drug

Study Arms (1)

RAD001

EXPERIMENTAL

Drug: everolimus (RAD001)

Interventions

everolimus (RAD001) DRUG

Subjects will resume the dosing regimen that they were receiving at the completion of the initial RAD001 study.

Also known as: RAD001

RAD001

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Novartis: collaborator

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Tuberous Sclerosis; Angiolipoma

Interventions

Everolimus

Condition Hierarchy (Ancestors)

Hamartoma; Neoplasms; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Nervous System Diseases; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Neoplasms, Adipose Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type

Intervention Hierarchy (Ancestors)

Sirolimus; Macrolides; Lactones; Organic Chemicals

Study Officials

• John Bissler, MD

  LeBonheur Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2008
• First Posted: November 18, 2008
• Study Start: December 1, 2008
• Primary Completion: September 1, 2013
• Study Completion: September 1, 2013
• Last Updated: September 18, 2013

Record last verified: 2013-09

Locations

US (1)