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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03826628




Registration number
NCT03826628
Ethics application status
Date submitted
29/01/2019
Date registered
1/02/2019
Date last updated
13/08/2019

Titles & IDs
Public title
Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex
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
Secondary ID [1] 0 0
DSLP-01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Facial Angiofibroma 0 0
Tuberous Sclerosis 0 0
Condition category
Condition code
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above
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 - 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

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of participants obtaining successful treatment - 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
Timepoint [1] 0 0
After 26 weeks treatment
Secondary outcome [1] 0 0
Time to treatment success - The time elapsed from the first dose to the time of treatment success, according to the IGA scale
Timepoint [1] 0 0
From first dose to 26 weeks
Secondary outcome [2] 0 0
Change from baseline in IGA - The change in grading on the IGA scale from baseline
Timepoint [2] 0 0
At baseline and after 26 weeks treatment
Secondary outcome [3] 0 0
Change from baseline in FASI - The change in grading on the Facial Angiofibroma Severity Index (FASI) from baseline
Timepoint [3] 0 0
At baseline and after 26 weeks treatment
Secondary outcome [4] 0 0
Subjective (participant or parent/caregiver) improvement rating - Percentage improvement in FA since beginning treatment, as assessed by the participant or parent/caregiver
Timepoint [4] 0 0
After 26 weeks treatment
Secondary outcome [5] 0 0
Objective (clinician) improvement rating - Percentage improvement in FA since beginning treatment, as assessed by the clinician
Timepoint [5] 0 0
After 26 weeks treatment
Secondary outcome [6] 0 0
Categorical Change in FA - Change in FA since beginning treatment on a 5-point scale, as assessed by the participant or parent/caregiver
Timepoint [6] 0 0
After 26 weeks treatment

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
Minimum age
6 Years
Maximum age
65 Years
Gender
Both males and females
Can healthy volunteers participate?
No
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

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/Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Children's Health Queensland - Brisbane
Recruitment postcode(s) [1] 0 0
4101 - Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Michigan
Country [3] 0 0
United States of America
State/province [3] 0 0
Minnesota
Country [4] 0 0
New Zealand
State/province [4] 0 0
Canterbury
Country [5] 0 0
Spain
State/province [5] 0 0
Navarra

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Dermatology Specialties Limited Partnership
Address
Country

Ethics approval
Ethics application status

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).
Trial website
https://clinicaltrials.gov/show/NCT03826628
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ioana Stanescu
Address 0 0
Dermatology Specialties Limited Partnership
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Jennifer Zhang
Address 0 0
Country 0 0
Phone 0 0
+ 64 9 488 0232
Fax 0 0
Email 0 0
jenniferz@aftpharm.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03826628