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

Dermatology Specialties

Status and phase: Completed; Phase 3; Phase 2

Conditions: Tuberous Sclerosis; Facial Angiofibroma

Treatments: Drug: rapamycin; Drug: placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03826628

DSLP-01

Details and patient eligibility

About

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).

Full description

Topical rapamycin has previously been used to treat FA associated with TSC, reducing erythema, papule size, while flattening lesions and improving skin texture. Topical rapamycin has been reported to be well tolerated.

The efficacy and safety of a topical rapamycin cream at two strengths (0.5% and 1.0%) will be assessed during a 26 week double-blind treatment phase with assessments made at clinical visits at baseline, 2, 8, 14, 20 and 26 weeks, and at follow-up (4 weeks after the last dose of study drug).

Enrollment: 107 patients

Sex: All

Ages: 6 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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107 participants in 3 patient groups, including a placebo group

0.5% Rapamycin cream, topical

Experimental group

Description:

Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks

Treatment:

Drug: rapamycin

1.0% Rapamycin cream, topical

Experimental group

Description:

Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks

Treatment:

Drug: rapamycin

Placebo

Placebo Comparator group

Description:

Placebo cream topical, applied once daily before bed on affected area for 26 weeks

Treatment:

Drug: placebo