Topical Rapamycin to Erase Angiofibromas in TSC

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Brief Title

Topical Rapamycin to Erase Angiofibromas in TSC

Official Title

Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy

Brief 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.

Study Phase

Phase 2

Study Type


Primary Outcome

Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score

Secondary Outcome

 Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient





Study Arms / Comparison Groups

Description:  Cream only


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

May 2012

Completion Date

August 2014

Primary Completion Date

August 2014

Eligibility 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.




N/A - N/A

Accepts Healthy Volunteers



Mary Kay Koenig, MD, , 

Location Countries


Location Countries


Administrative Informations



Organization ID


Secondary IDs

Department of Defense USAMRMC

Responsible Party

Principal Investigator

Study Sponsor

The University of Texas Health Science Center, Houston

Study Sponsor

Mary Kay Koenig, MD, Principal Investigator, The University of Texas Medical School at Houston

Verification Date

September 2020