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

September 17, 2013 updated by: Children's Hospital Medical Center, Cincinnati

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

20

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: RAD001
Drug: everolimus (RAD001)
Subjects will resume the dosing regimen that they were receiving at the completion of the initial RAD001 study.
Other Names:
  • RAD001

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
RAD001 tolerance
Time Frame: Every three months while on study drug
Every three months while on study drug

Secondary Outcome Measures

Outcome Measure
Time Frame
Angiomyolipoma volume reduction
Time Frame: Every year while on study drug
Every year while on study drug

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's Hospital Medical Center, Cincinnati

Collaborators

Novartis

Investigators

  • Principal Investigator: John Bissler, MD, LeBonheur Children's Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2008

Primary Completion (ACTUAL)

September 1, 2013

Study Completion (ACTUAL)

September 1, 2013

Study Registration Dates

First Submitted

November 17, 2008

First Submitted That Met QC Criteria

November 17, 2008

First Posted (ESTIMATE)

November 18, 2008

Study Record Updates

Last Update Posted (ESTIMATE)

September 18, 2013

Last Update Submitted That Met QC Criteria

September 17, 2013

Last Verified

September 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CCHMC IRB# 2008-0333

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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