Register a trial

Please note the ANZCTR will be unattended from Friday 20 December 2024 for the holidays. The Registry will re-open on Tuesday 7 January 2025. Submissions and updates will not be processed during that time.

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03826628
Ethics application status
Date submitted
29/01/2019
Date registered
1/02/2019

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
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
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

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
Timepoint [1] 0 0
After 26 weeks treatment
Secondary outcome [1] 0 0
Time to Treatment Success
Timepoint [1] 0 0
From first dose to 26 weeks (± 2 weeks)
Secondary outcome [2] 0 0
Change From Baseline in Investigator's Global Assessment
Timepoint [2] 0 0
At baseline and after 26 weeks treatment
Secondary outcome [3] 0 0
Change From Baseline in Facial Angiofibroma Severity Index (FASI)
Timepoint [3] 0 0
At baseline and after 26 weeks treatment
Secondary outcome [4] 0 0
Subjective (Participant or Parent/Caregiver) Percentage Change Rating Scale
Timepoint [4] 0 0
After 26 weeks treatment
Secondary outcome [5] 0 0
Objective (Clinician) Percentage Change Rating Scale
Timepoint [5] 0 0
After 26 weeks treatment
Secondary outcome [6] 0 0
Categorical Change in Facial Angiofibroma
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
Sex
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
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
28/07/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/09/2022
Sample size
Target
Accrual to date
Final
107
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
Arizona
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
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Michigan
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
Virginia
Country [7] 0 0
Czechia
State/province [7] 0 0
Brno
Country [8] 0 0
Hungary
State/province [8] 0 0
Budapest
Country [9] 0 0
Hungary
State/province [9] 0 0
Pécs
Country [10] 0 0
New Zealand
State/province [10] 0 0
Canterbury
Country [11] 0 0
Serbia
State/province [11] 0 0
Belgrade
Country [12] 0 0
Slovakia
State/province [12] 0 0
Bratislava
Country [13] 0 0
Spain
State/province [13] 0 0
Navarra
Country [14] 0 0
Spain
State/province [14] 0 0
Madrid
Country [15] 0 0
Taiwan
State/province [15] 0 0
Taipei

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Individual Participant Data is not planned to be shared with other researchers


What supporting documents are/will be available?




Results publications and other study-related documents

TypeCitations or Other Details
Journal Aitken P, Stanescu I, Boddington L, Mahon C, Fogar... [More Details]


Results are available at https://clinicaltrials.gov/study/NCT03826628