Back to resultsLong Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)
Primary Purpose
Tuberous Sclerosis, Angiolipoma
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
everolimus (RAD001)
Sponsored by
About this trial
This is an interventional treatment trial for Tuberous Sclerosis focused on measuring Tuberous Sclerosis Complex (TSC), Angiolipoma, mTOR, Everolimus
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.
Sites / Locations
- Cincinnati Children's Hospital Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
RAD001
Arm Description
Outcomes
Primary Outcome Measures
RAD001 tolerance
Secondary Outcome Measures
Angiomyolipoma volume reduction
Full Information
NCT ID
NCT00792766
First Posted
November 17, 2008
Last Updated
September 17, 2013
Sponsor
Children's Hospital Medical Center, Cincinnati
Collaborators
Novartis
1. Study Identification
Unique Protocol Identification Number
NCT00792766
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
Study Type
Interventional
2. Study Status
Record Verification Date
September 2013
Overall Recruitment Status
Completed
Study Start Date
December 2008 (undefined)
Primary Completion Date
September 2013 (Actual)
Study Completion Date
September 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Hospital Medical Center, Cincinnati
Collaborators
Novartis
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Tuberous Sclerosis, Angiolipoma
Keywords
Tuberous Sclerosis Complex (TSC), Angiolipoma, mTOR, Everolimus
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Actual)
8. Arms, Groups, and Interventions
Arm Title
RAD001
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
everolimus (RAD001)
Other Intervention Name(s)
RAD001
Intervention Description
Subjects will resume the dosing regimen that they were receiving at the completion of the initial RAD001 study.
Primary Outcome Measure Information:
Title
RAD001 tolerance
Time Frame
Every three months while on study drug
Secondary Outcome Measure Information:
Title
Angiomyolipoma volume reduction
Time Frame
Every year while on study drug
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
John Bissler, MD
Organizational Affiliation
LeBonheur Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)
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