Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

• Register: ClinicalTrials.gov
• Last refreshed on: 21 August 2023
• Main ID: NCT03826628
• Date of registration: 29/01/2019
• Prospective Registration: Yes
• Primary sponsor: Dermatology Specialties Limited Partnership
• 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
• Date of first enrolment: July 28, 2019
• Target sample size: 107
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03826628
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2/Phase 3

Countries of recruitment

Australia, Czechia, Hungary, New Zealand, Serbia, Slovakia, Spain, Taiwan, United Kingdom, United States

Contacts

• Name: Ioana Stanescu
• Affiliation: Dermatology Specialties Limited Partnership

Key inclusion & exclusion criteria

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

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



Age minimum: 6 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Facial Angiofibroma

Tuberous Sclerosis

Intervention(s)

Drug: placebo

Drug: rapamycin

Primary Outcome(s)

Percentage of Participants Obtaining Successful Treatment [Time Frame: After 26 weeks treatment]

Secondary Outcome(s)

Subjective (Participant or Parent/Caregiver) Percentage Change Rating Scale [Time Frame: After 26 weeks treatment]

Objective (Clinician) Percentage Change Rating Scale [Time Frame: After 26 weeks treatment]

Time to Treatment Success [Time Frame: From first dose to 26 weeks]

Change From Baseline in Investigator's Global Assessment [Time Frame: At baseline and after 26 weeks treatment]

Categorical Change in Facial Angiofibroma [Time Frame: After 26 weeks treatment]

Change From Baseline in Facial Angiofibroma Severity Index (FASI) [Time Frame: At baseline and after 26 weeks treatment]

Secondary ID(s)

DSLP-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 14/08/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03826628