Phase 3
N=107
Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex
Facial Angiofibroma · Tuberous Sclerosis
Bottom Line
View on ClinicalTrials.gov: NCT03826628 ↗Enrolled (actual)
107
Serious AEs
5.6%
Results posted
Aug 2023
Primary outcome: Primary: Percentage of Participants Obtaining Successful Treatment — 4; 3; 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- rapamycin (Drug); placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 6+ yrs
- Sex
- All
- Sponsor
- Dermatology Specialties Limited Partnership
- Primary completion
- Sep 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants Obtaining Successful Treatment |
4; 3; 2 | — |
| SECONDARY Time to Treatment Success |
25.89; 26.71; 19.7 | — |
| SECONDARY Change From Baseline in Investigator's Global Assessment |
4; 3; 2; 16; 17; 7 | — |
| SECONDARY Change From Baseline in Facial Angiofibroma Severity Index (FASI) |
1; 1; 0; 1; 3; 1 | — |
| SECONDARY Subjective (Participant or Parent/Caregiver) Percentage Change Rating Scale |
57.06; 54.06; 30.84 | — |
| SECONDARY Objective (Clinician) Percentage Change Rating Scale |
47.83; 49.39; 20.76 | — |
| SECONDARY Categorical Change in Facial Angiofibroma |
1; 0; 0; 0; 1; 12 | — |
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).
Eligibility Criteria
Inclusion Criteria
- Male and female patients aged ≥ 6 years and ≤ 65 years on the day informed consent is obtained
- Patients diagnosed with TSC based on the clinical diagnostic criteria of International Tuberous Sclerosis Complex Consensus Conference 2012 and presenting visible facial angiofibroma
- An FA severity score of 2 or 3 on the IGA scale
- Patients or their legal representatives capable of understanding the explanation of the clinical trial and who give written informed consent for participation
- Patients or their legal representatives able to maintain patient diaries following the instructions of the investigator or sub-investigator
Exclusion Criteria
- Patients who cannot carry out the treatment plan or follow-up assessment
- Patients with serious skin lesions such as erosions or ulcers
- Patients with known hypersensitivity to any component of the study product
- Patients who have received rapamycin/sirolimus, everolimus, or temsirolimus within 3 months of enrolment
- Patients who received laser therapy or surgical therapy within 6 months prior to trial enrolment
- Patients who participated in any other clinical trial within 3 months prior to the day of enrolment
- Patients judged unsuitable for this clinical trial by the investigator or sub-investigator
- Pregnant or lactating females
- Sexually active females of childbearing potential not using adequate contraception and sexually active males not using adequate contraception
- Patients with immune dysfunction or receiving any form of immunosuppression
- Patients with severe FA, with a score of 4 on the IGA scale
- Patients with an FA severity score of less than 2 on the IGA scale
Data sourced from ClinicalTrials.gov (NCT03826628). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.