Topical Rapamycin to Erase Angiofibromas in TSC

Tuberous Sclerosis Clinical Trial

— Treatment  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01526356
• Other study ID #: HSC-MS-11-0501
• Secondary ID: Department of De
• Status: Completed
• Phase: Phase 2
• Start date: May 2012
• Est. completion date: August 2014

Study information

• Verified date: September 2020
• Source: The University of Texas Health Science Center, Houston
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 179
• Est. completion date: August 2014
• Est. primary completion date: August 2014
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Angiofibroma
• Angiofibromas
• Sclerosis
• Tuberous Sclerosis

Intervention

Drug:
• 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

Locations

Country	Name	City	State
Australia	Sydney Children's Hospital	Sydney	New South Wales
United States	Kennedy Krieger Institute	Baltimore	Maryland
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Herscot Center for Adults and Children with TSC Massachusetts General Hospital	Boston	Massachusetts
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	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	Clinic Without Walls	Saint Paul	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
The University of Texas Health Science Center, Houston

Countries where clinical trial is conducted

United States,  Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Number of Events of Dermatologic Sensitivity at the Site of Application	Dermatologic sensitivity includes pain, pruritis, or erythema at the application site.	6 months
Other	Number of Participants With Systemic Uptake of Topically Applied Rapamycin	Blood levels checked to confirm the lack of systemic rapamycin.	6 months
Primary	Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score	Subject's lesion/s will be digitally photographed before initial application (visit 1), at each study visit (visits 2 through 6), and immediately prior to study termination (visit 7). Following completion of the study, all photographs will be evaluated by two independent dermatologists blinded to both the treatment arm and the stage of treatment. The dermatologists will assess each photograph's appearance using the facial Angiofibroma Grading Scale (AGS), which assesses the forehead, nose, cheeks, and chin for erythema, average lesion size, lesion density, and percent involvement, as well as presence of any pedunculated angiofibromas. A total score is reported with a range of 0 to 202, with higher scores representing a worse outcome.	baseline, 6 months
Secondary	Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient		baseline, 6 months
Secondary	Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI)	The Dermatology life Quality Index (DLQI) is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. It is designed for people aged 16 years and above. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).	baseline, 6 months
Secondary	Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI)	The Children's Dermatology life Quality Index (DLQI) is validated from the age of four years to 16 years. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).	baseline, 6 months
Secondary	Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI)	The Family Dermatology life Quality Index (FLQI) is designed for adult (more than 16 years of age) family members or partners of patients (of any age) with any skin disease. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).	baseline, 6 months