Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
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COMPLETED
PHASE2
179 participants
INTERVENTIONAL
2012-05-31
2014-08-31
Brief Summary
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The primary goal of this study is to evaluate the efficacy of the topical medication for reduction of cutaneous angiofibromas in patients with TSC. The secondary goal of this study is to confirm the safety of the topical medication.
Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Placebo
Cream only
Placebo
Study cream is applied nightly to the affected areas on the face.
0.1 % Rapamycin
0.1% Rapamycin cream
Rapamycin
Study cream is applied nightly to the affected areas on the face. Low Dose
1% Rapamycin
1% Rapamycin cream
Rapamycin
Study cream is applied nightly to the affected areas on the face. High Dose
Interventions
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Placebo
Study cream is applied nightly to the affected areas on the face.
Rapamycin
Study cream is applied nightly to the affected areas on the face. Low Dose
Rapamycin
Study cream is applied nightly to the affected areas on the face. High Dose
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Subject has a diagnosis of TSC and has visible facial angiofibromas.
* Female subjects of child bearing potential must not be pregnant and must agree to use appropriate contraceptive methods .
Exclusion Criteria
* Subject is receiving any form of immunosuppression or has previously experienced immune dysfunction.
* Subject is currently participating in or has participated within the last 30 days in a clinical trial involving an investigational drug.
* Subject has a known hypersensitivity to either the vehicle or Rapamycin.
* Subject is a pregnant or nursing female.
* Subject has other dermatologic conditions that would preclude or prevent adequate assessment of changes to their facial angiofibromas.
* Subject has had laser surgery, cryotherapy, or other dermatologic treatment to their facial angiofibromas within the previous 6 months.
ALL
No
Sponsors
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The University of Texas Health Science Center, Houston
OTHER
Responsible Party
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Mary Kay Koenig
Associate Professor
Principal Investigators
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Mary Kay Koenig, MD
Role: PRINCIPAL_INVESTIGATOR
The University of Texas Medical School at Houston
Hope Northrup, MD
Role: PRINCIPAL_INVESTIGATOR
The University of Texas Medical School at Houston
Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
UCLA Mattel Children's Hospital
Los Angeles, California, United States
Jack & Julia Center for TSC at Oakland Children's Hospital & Research Center
Oakland, California, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
Herscot Center for Adults and Children with TSC Massachusetts General Hospital
Boston, Massachusetts, United States
Clinic Without Walls
Saint Paul, Minnesota, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Texas Scottish Rite Hospital
Dallas, Texas, United States
The University of Texas Medical School at Houston
Houston, Texas, United States
Sydney Children's Hospital
Sydney, New South Wales, Australia
Countries
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References
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Koenig MK, Bell CS, Hebert AA, Roberson J, Samuels JA, Slopis JM, Tate P, Northrup H; TREATMENT Trial Collaborators. Efficacy and Safety of Topical Rapamycin in Patients With Facial Angiofibromas Secondary to Tuberous Sclerosis Complex: The TREATMENT Randomized Clinical Trial. JAMA Dermatol. 2018 Jul 1;154(7):773-780. doi: 10.1001/jamadermatol.2018.0464.
Other Identifiers
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Department of Defense USAMRMC
Identifier Type: OTHER_GRANT
Identifier Source: secondary_id
HSC-MS-11-0501
Identifier Type: -
Identifier Source: org_study_id