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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

Page last updated: August 23, 2015

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