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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01526356
Registration number
NCT01526356
Ethics application status
Date submitted
1/02/2012
Date registered
3/02/2012
Date last updated
26/10/2020
Titles & IDs
Public title
Topical Rapamycin to Erase Angiofibromas in TSC
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Scientific title
Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy
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Secondary ID [1]
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Department of Defense USAMRMC
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Secondary ID [2]
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HSC-MS-11-0501
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Universal Trial Number (UTN)
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Trial acronym
Treatment
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Angiofibromas
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Tuberous Sclerosis
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Other neurological disorders
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Skin
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Dermatological conditions
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Skin
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Other skin conditions
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Cardiovascular
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Other cardiovascular diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Placebo
Treatment: Drugs - Rapamycin
Treatment: Drugs - Rapamycin
Placebo Comparator: Placebo - Cream only
Active Comparator: 0.1 % Rapamycin - 0.1% Rapamycin cream
Active Comparator: 1% Rapamycin - 1% Rapamycin cream
Treatment: Drugs: Placebo
Study cream is applied nightly to the affected areas on the face.
Treatment: Drugs: Rapamycin
Study cream is applied nightly to the affected areas on the face. Low Dose
Treatment: Drugs: Rapamycin
Study cream is applied nightly to the affected areas on the face. High Dose
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score
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Assessment method [1]
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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.
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Timepoint [1]
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baseline, 6 months
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Secondary outcome [1]
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Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient
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Assessment method [1]
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Timepoint [1]
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baseline, 6 months
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Secondary outcome [2]
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Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI)
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Assessment method [2]
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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).
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Timepoint [2]
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baseline, 6 months
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Secondary outcome [3]
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Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI)
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Assessment method [3]
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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).
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Timepoint [3]
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baseline, 6 months
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Secondary outcome [4]
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Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI)
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Assessment method [4]
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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).
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Timepoint [4]
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baseline, 6 months
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Eligibility
Key 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 .
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/05/2012
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/08/2014
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Sample size
Target
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Accrual to date
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Final
179
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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Sydney Children's Hospital - Sydney
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Recruitment postcode(s) [1]
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- Sydney
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Alabama
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Country [2]
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United States of America
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State/province [2]
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California
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Country [3]
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United States of America
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State/province [3]
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Maryland
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Country [4]
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United States of America
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State/province [4]
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Massachusetts
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Country [5]
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United States of America
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State/province [5]
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Minnesota
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Country [6]
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United States of America
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State/province [6]
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Ohio
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Country [7]
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United States of America
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State/province [7]
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Texas
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Funding & Sponsors
Primary sponsor type
Other
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Name
The University of Texas Health Science Center, Houston
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01526356
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Mary Kay Koenig, MD
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Address
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The University of Texas Medical School at Houston
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01526356
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