Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
Scientific title
Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy
Secondary ID [1] 0 0
Department of Defense USAMRMC
Secondary ID [2] 0 0
HSC-MS-11-0501
Universal Trial Number (UTN)
Trial acronym
Treatment
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Angiofibromas 0 0
Tuberous Sclerosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Skin 0 0 0 0
Dermatological conditions
Skin 0 0 0 0
Other skin conditions
Cardiovascular 0 0 0 0
Other cardiovascular diseases

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score
Timepoint [1] 0 0
baseline, 6 months
Secondary outcome [1] 0 0
Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient
Timepoint [1] 0 0
baseline, 6 months
Secondary outcome [2] 0 0
Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI)
Timepoint [2] 0 0
baseline, 6 months
Secondary outcome [3] 0 0
Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI)
Timepoint [3] 0 0
baseline, 6 months
Secondary outcome [4] 0 0
Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI)
Timepoint [4] 0 0
baseline, 6 months

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 .
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/05/2012
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/08/2014
Sample size
Target
Accrual to date
Final
179
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Sydney Children's Hospital - Sydney
Recruitment postcode(s) [1] 0 0
- Sydney
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas

Funding & Sponsors
Primary sponsor type
Other
Name
The University of Texas Health Science Center, Houston
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Mary Kay Koenig, MD
Address 0 0
The University of Texas Medical School at Houston
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT01526356