Back to resultsRapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM
Primary Purpose
Tuberous Sclerosis, Lymphangioleiomyomatosis
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Rapamycin, sirolimus
Sponsored by
About this trial
This is an interventional treatment trial for Tuberous Sclerosis focused on measuring TSC and LAM, Tuberous Sclerosis Complex and sporadic LAM
Eligibility Criteria
Inclusion Criteria:
- Diagnosed with angiomyolipomas and have either Tuberous Sclerosis complex or Lymphangioleiomyomatosis
- Between the gaes of 18 and 65 years
- Competency to voluntarily consent
- Clinically definite diagnosis of tuberous sclerosis or S-LAM
- Adequate contraception
- At least one angiomyolipoma of 1 cm or greater in largest diameter
Exclusion Criteria:
- Use of continuous supplemental oxygen
- Concurrent infection
- Recent surgery
- Ongoing or planned pregnancy
- Lactation
- Use of an investigational drug within the last 30 days of study entrance
Sites / Locations
- Cincinnati Childrens Hospital Medical Center
Outcomes
Primary Outcome Measures
Volume of Angiomyolipomata
Secondary Outcome Measures
Full Information
NCT ID
NCT00457808
First Posted
April 6, 2007
Last Updated
April 6, 2007
Sponsor
Children's Hospital Medical Center, Cincinnati
Collaborators
The LAM Foundation, Tuberous Sclerosis Alliance
1. Study Identification
Unique Protocol Identification Number
NCT00457808
Brief Title
Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM
Official Title
Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
Study Type
Interventional
2. Study Status
Record Verification Date
April 2007
Overall Recruitment Status
Completed
Study Start Date
December 2002 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
March 2006 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
Children's Hospital Medical Center, Cincinnati
Collaborators
The LAM Foundation, Tuberous Sclerosis Alliance
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.
Detailed Description
The study design was an open label, phase I/II trial of sirolimus for one year followed by one year off therapy.Patients were seen at baseline,at two weeks to four weeks, and at 2,4,6,9,12,18 and 24 months. Angiomyolipomata imaging was performed at all but the two to four week visit. Complete pulmonary function tests and six-minute walk were obtained at baseline,6 or 9 month, 12 and 24 month visits, while simple spirometry only was performed at all other visits.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Tuberous Sclerosis, Lymphangioleiomyomatosis
Keywords
TSC and LAM, Tuberous Sclerosis Complex and sporadic LAM
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
25 (false)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Rapamycin, sirolimus
Primary Outcome Measure Information:
Title
Volume of Angiomyolipomata
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:
Diagnosed with angiomyolipomas and have either Tuberous Sclerosis complex or Lymphangioleiomyomatosis
Between the gaes of 18 and 65 years
Competency to voluntarily consent
Clinically definite diagnosis of tuberous sclerosis or S-LAM
Adequate contraception
At least one angiomyolipoma of 1 cm or greater in largest diameter
Exclusion Criteria:
Use of continuous supplemental oxygen
Concurrent infection
Recent surgery
Ongoing or planned pregnancy
Lactation
Use of an investigational drug within the last 30 days of study entrance
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
John Bissler, MD
Organizational Affiliation
Cincinnati Childrens Hospital Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cincinnati Childrens Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229-3039
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
18184959
Citation
Bissler JJ, McCormack FX, Young LR, Elwing JM, Chuck G, Leonard JM, Schmithorst VJ, Laor T, Brody AS, Bean J, Salisbury S, Franz DN. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med. 2008 Jan 10;358(2):140-51. doi: 10.1056/NEJMoa063564.
Results Reference
derived
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Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM
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