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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/NCT03826628
Registration number
NCT03826628
Ethics application status
Date submitted
29/01/2019
Date registered
1/02/2019
Date last updated
8/09/2023
Titles & IDs
Public title
Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex
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Scientific title
A Phase 2/3, Multi-Center, Double-Blind, Placebo-Controlled, Randomized, Parallel-Group, Dose-Response Comparison of the Efficacy and Safety of a Topical Rapamycin Cream for the Treatment of Facial Angiofibromas (FA) Associated With Tuberous Sclerosis Complex (TSC) in Patients 6 Years of Age and Over
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Secondary ID [1]
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DSLP-01
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Facial Angiofibroma
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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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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - rapamycin
Treatment: Drugs - placebo
Experimental: 0.5% Rapamycin cream, topical - Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks
Experimental: 1.0% Rapamycin cream, topical - Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks
Placebo Comparator: Placebo - Placebo cream topical, applied once daily before bed on affected area for 26 weeks
Treatment: Drugs: rapamycin
Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks
Treatment: Drugs: placebo
Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks
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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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Percentage of Participants Obtaining Successful Treatment
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Assessment method [1]
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Success on the Investigator Global Assessment (IGA) scale is defined as clear or almost clear with an improvement of at least two grades from baseline. IGA scores range from 0-4:
0=Clear
Almost Clear
Mild
Moderate
Severe
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Timepoint [1]
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After 26 weeks treatment
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Secondary outcome [1]
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Time to Treatment Success
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Assessment method [1]
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The time elapsed from the first dose to the time of treatment success, according to the Investigator's Global Assessment (IGA) scale. The total time of treatment was 26 weeks, although Covid-19 visit delays led to an extension of up to 2 weeks (28 weeks total) for some patients. Success on the Investigator Global Assessment (IGA) scale is defined as clear or almost clear with an improvement of at least two grades from baseline. IGA scores range from 0-4:
0=Clear
Almost Clear
Mild
Moderate
Severe
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Timepoint [1]
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From first dose to 26 weeks (± 2 weeks)
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Secondary outcome [2]
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Change From Baseline in Investigator's Global Assessment
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Assessment method [2]
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The change in grading on the Investigator's Global Assessment (IGA) scale from baseline.
IGA scores range from 0-4:
0=Clear
Almost Clear
Mild
Moderate
Severe
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Timepoint [2]
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At baseline and after 26 weeks treatment
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Secondary outcome [3]
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Change From Baseline in Facial Angiofibroma Severity Index (FASI)
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Assessment method [3]
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The change in grading on the Facial Angiofibroma Severity Index (FASI) from baseline. FASI grades lesions according to their erythema, size and extent by summing the scores of each category. The final FASI scores range from (mild) 2-9 (severe).
Erythema Skin color 0 Light Red 1 Red 2 Dark Red/purple 3 Size None 0 Small (< 5mm) 1 Large (> 5mm) 2 Confluent 3 Extension <50 % cheek surface 2 >50% cheek surface 3
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Timepoint [3]
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At baseline and after 26 weeks treatment
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Secondary outcome [4]
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Subjective (Participant or Parent/Caregiver) Percentage Change Rating Scale
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Assessment method [4]
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Percentage change in facial angiofibroma since beginning treatment, as assessed by the participant or parent/caregiver. A large value indicates most improvement to facial angiofibroma (minimum=0, maximum=100). This was a single assessment time-point, where the participant or parent/caregiver estimated the percentage change in the facial angiofibroma lesion appearance from their perspective since baseline.
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Timepoint [4]
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After 26 weeks treatment
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Secondary outcome [5]
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Objective (Clinician) Percentage Change Rating Scale
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Assessment method [5]
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Percentage improvement in facial angiofibroma since beginning treatment, as assessed by the clinician. A large value indicates most improvement to facial angiofibroma (minimum=0, maximum=100). This was a single assessment time-point, where clinicians estimated the percentage change in the facial angiofibroma lesion appearance from their perspective since baseline.
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Timepoint [5]
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After 26 weeks treatment
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Secondary outcome [6]
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Categorical Change in Facial Angiofibroma
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Assessment method [6]
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Change in facial angiofibroma since beginning treatment on a 5-point scale, as assessed by the participant or parent/caregiver. This was a single assessment time-point, where the participant or parent/caregiver evaluated the change in the facial angiofibroma lesion appearance from their perspective since baseline.
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Timepoint [6]
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After 26 weeks treatment
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Eligibility
Key inclusion criteria
1. Male and female patients aged = 6 years and = 65 years on the day informed consent is
obtained
2. Patients diagnosed with TSC based on the clinical diagnostic criteria of International
Tuberous Sclerosis Complex Consensus Conference 2012 and presenting visible facial
angiofibroma
3. An FA severity score of 2 or 3 on the IGA scale
4. Patients or their legal representatives capable of understanding the explanation of
the clinical trial and who give written informed consent for participation
5. Patients or their legal representatives able to maintain patient diaries following the
instructions of the investigator or sub-investigator
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Minimum age
6
Years
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Maximum age
65
Years
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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
1. Patients who cannot carry out the treatment plan or follow-up assessment
2. Patients with serious skin lesions such as erosions or ulcers
3. Patients with known hypersensitivity to any component of the study product
4. Patients who have received rapamycin/sirolimus, everolimus, or temsirolimus within 3
months of enrolment
5. Patients who received laser therapy or surgical therapy within 6 months prior to trial
enrolment
6. Patients who participated in any other clinical trial within 3 months prior to the day
of enrolment
7. Patients judged unsuitable for this clinical trial by the investigator or
sub-investigator
8. Pregnant or lactating females
9. Sexually active females of childbearing potential not using adequate contraception and
sexually active males not using adequate contraception
10. Patients with immune dysfunction or receiving any form of immunosuppression
11. Patients with severe FA, with a score of 4 on the IGA scale
12. Patients with an FA severity score of less than 2 on the IGA scale
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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/Phase 3
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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
28/07/2019
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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
1/09/2022
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Sample size
Target
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Accrual to date
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Final
107
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Recruitment in Australia
Recruitment state(s)
QLD
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Recruitment hospital [1]
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Children's Health Queensland - Brisbane
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Recruitment postcode(s) [1]
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4101 - Brisbane
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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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Arizona
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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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Florida
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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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Michigan
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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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Virginia
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Country [7]
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Czechia
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State/province [7]
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Brno
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Country [8]
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Hungary
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State/province [8]
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Budapest
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Country [9]
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Hungary
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State/province [9]
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Pécs
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Country [10]
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New Zealand
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State/province [10]
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Canterbury
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Country [11]
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Serbia
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State/province [11]
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Belgrade
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Country [12]
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Slovakia
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State/province [12]
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Bratislava
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Country [13]
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Spain
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State/province [13]
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Navarra
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Country [14]
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Spain
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State/province [14]
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Madrid
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Country [15]
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Taiwan
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State/province [15]
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Taipei
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Dermatology Specialties Limited Partnership
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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 aims to compare the safety and efficacy of two different strengths of Rapamycin
cream, topical and placebo over 26 weeks in the treatment of facial angiofibroma (FA)
associated with Tuberous Sclerosis Complex (TSC).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03826628
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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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Ioana Stanescu
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Address
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Dermatology Specialties Limited Partnership
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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/NCT03826628
Download to PDF