Clinical trialNCT01526356CompletedPhase 2Interventional

Topical Rapamycin to Erase Angiofibromas in TSC

SponsorThe University of Texas Health Science Center, HoustonEnrollment179AngiofibromasTuberous Sclerosis

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.

clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Hope Northrup, MD
The University of Texas Medical School at Houston
Principal Investigator
clinicaltrials.gov ↗
Mary Kay Koenig, MD
The University of Texas Medical School at Houston
Principal Investigator
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

10 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

Australia

Sydney Children'S Hospital
Sydney, New South Wales, Australia

United States

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

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

PlaceboSirolimus →

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 31 May 2012
Primary completion: 31 Aug 2014
Completion: 31 Aug 2014
Last update posted: 26 Oct 2020
Registered enrollment: 179
Registry id: NCT01526356

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.