Topical Rapamycin to Erase Angiofibromas in TSC
Information source: The University of Texas Health Science Center, Houston
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Angiofibromas; Tuberous Sclerosis
Intervention: Placebo (Drug); Rapamycin (Drug); Rapamycin (Drug)
Phase: Phase 2
Status: Completed
Sponsored by: The University of Texas Health Science Center, Houston Official(s) and/or principal investigator(s): Mary Kay Koenig, MD, Principal Investigator, Affiliation: The University of Texas Medical School at Houston Hope Northrup, MD, Principal Investigator, Affiliation: The University of Texas Medical School at Houston
Summary
The study is a multi-center prospective, randomized, double-blind, placebo-controlled
evaluation of the safety and efficacy of a topically applied formulation of rapamycin to
cutaneous angiofibromas in subjects with Tuberous Sclerosis Complex (TSC). Subjects will
apply either the topical vehicle containing rapamycin or the topical vehicle alone nightly
to their angiofibromas for six months.
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.
Clinical Details
Official title: Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary outcome: Reduction in lesion size and appearance
Secondary outcome: Confirm the lack of systemic uptake of topically applied rapamycin.Dermatologic sensitivity at the site of application.
Eligibility
Minimum age: N/A.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Subjects must be willing and able to comply with all trial requirements.
- 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 currently receiving therapy with Rapamycin.
- 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.
Locations and Contacts
University of Alabama at Birmingham, Birminham, Alabama 35294, United States
UCLA Mattel Children's Hospital, Los Angeles, California 90095, United States
Jack & Julia Center for TSC at Oakland Children's Hospital & Research Center, Oakland, California 94609, United States
Kennedy Krieger Institute, Baltimore, Maryland 21205, United States
Herscot Center for Adults and Children with TSC Massachusetts General Hospital, Boston, Massachusetts 02114, United States
Clinic Without Walls, Saint Paul, Minnesota 55102-2697, United States
Sydney Children's Hospital, Sydney, New South Wales, Australia
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio 45229, United States
Texas Scottish Rite Hospital, Dallas, Texas 75219, United States
The University of Texas Medical School at Houston, Houston, Texas 77030, United States
Additional Information
Starting date: May 2012
Last updated: June 9, 2015
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