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

Sponsors and Collaborators:	Children's Hospital Medical Center, Cincinnati Novartis
Information provided by:	Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:	NCT00792766

Purpose

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.

Condition	Intervention	Phase
Tuberous Sclerosis Angiolipoma	Drug: everolimus (RAD001)	Phase I Phase II

Genetics Home Reference related topics: familial encephalopathy with neuroserpin inclusion bodies tuberous sclerosis

MedlinePlus related topics: Tuberous Sclerosis

Drug Information available for: Everolimus

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label, Single Group Assignment, Safety/Efficacy Study
Official Title:	Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

Further study details as provided by Children's Hospital Medical Center, Cincinnati:

Primary Outcome Measures:

RAD001 tolerance [ Time Frame: Every three months while on study drug ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Angiomyolipoma volume reduction [ Time Frame: Every year while on study drug ] [ Designated as safety issue: No ]

Estimated Enrollment:	10
Study Start Date:	December 2008
Estimated Study Completion Date:	June 2014
Estimated Primary Completion Date:	January 2014 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
RAD001: Experimental	Drug: everolimus (RAD001) Subjects will resume the dosing regimen that they were receiving at the completion of the initial RAD001 study.

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 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.

Eligibility

Ages Eligible for Study:	18 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00792766

Contacts

Contact: Elizabeth Coombs, RN

513-636-6274

elizabeth.coombs@cchmc.org

Locations

United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229

Sponsors and Collaborators

Children's Hospital Medical Center, Cincinnati

Novartis

Investigators

Principal Investigator:

John Bissler, MD

Children's Hospital Medical Center, Cincinnati

More Information

Responsible Party:	Cincinnati Children's Hospital Medical Center ( John Bissler, MD )
Study ID Numbers:	CCHMC IRB# 2008-0333
Study First Received:	November 17, 2008
Last Updated:	November 17, 2008
ClinicalTrials.gov Identifier:	NCT00792766
Health Authority:	United States: Food and Drug Administration

Keywords provided by Children's Hospital Medical Center, Cincinnati:

Tuberous Sclerosis Complex (TSC)
Angiolipoma
mTOR
Everolimus

Study placed in the following topic categories:

Everolimus
Immunoproliferative Disorders
Nervous System Malformations
Lymphangiomyoma
Sclerosis
Angiomyolipoma
Neurodegenerative Diseases
Angiolipoma
Bourneville syndrome
Lymphatic Diseases

Lymphangioleiomyomatosis
Neoplasms, Connective and Soft Tissue
Tuberous Sclerosis
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn
Tuberous sclerosis
Lymphoproliferative Disorders
Malformations of Cortical Development
Congenital Abnormalities
Neurocutaneous Syndromes

Additional relevant MeSH terms:

Lymphatic Vessel Tumors
Neoplasms
Neoplasms by Histologic Type
Pathologic Processes
Immunologic Factors
Immune System Diseases

Physiological Effects of Drugs
Nervous System Diseases
Immunosuppressive Agents
Hamartoma
Pharmacologic Actions
Neoplasms, Adipose Tissue

ClinicalTrials.gov processed this record on January 16, 2009