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

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Brief Title

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

Official Title

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

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.
    

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 Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

RAD001 tolerance

Secondary Outcome

 Angiomyolipoma volume reduction

Condition

Tuberous Sclerosis

Intervention

everolimus (RAD001)

Study Arms / Comparison Groups

 RAD001
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

20

Start Date

December 2008

Completion Date

September 2013

Primary Completion Date

September 2013

Eligibility 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.
      

Gender

All

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

John Bissler, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00792766

Organization ID

CCHMC IRB# 2008-0333


Responsible Party

Sponsor

Study Sponsor

Children's Hospital Medical Center, Cincinnati

Collaborators

 Novartis

Study Sponsor

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


Verification Date

September 2013