Trial record 8 of 35 for:    "tuberous sclerosis"

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
  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 1
Phase 2

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

Resource links provided by NLM:


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 ]

Enrollment: 20
Study Start Date: December 2008
Study Completion Date: September 2013
Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RAD001 Drug: everolimus (RAD001)
Subjects will resume the dosing regimen that they were receiving at the completion of the initial RAD001 study.
Other Name: RAD001

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

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 LeBonheur Children's Hospital
  More Information

No publications provided

Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT00792766     History of Changes
Other Study ID Numbers: CCHMC IRB# 2008-0333
Study First Received: November 17, 2008
Last Updated: September 17, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Hospital Medical Center, Cincinnati:
Tuberous Sclerosis Complex (TSC)
Angiolipoma
mTOR
Everolimus

Additional relevant MeSH terms:
Tuberous Sclerosis
Sclerosis
Angiolipoma
Angiomyolipoma
Lymphangioleiomyomatosis
Pathologic Processes
Hamartoma
Neoplasms
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Neoplasms, Adipose Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Perivascular Epithelioid Cell Neoplasms
Lymphangiomyoma
Lymphatic Vessel Tumors
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Everolimus
Sirolimus

ClinicalTrials.gov processed this record on September 14, 2014