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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
First received
Last updated
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
Health authority
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.

Study Design


Related Conditions & MeSH terms


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
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