Study to evaluate if the study medication, Rapamycin, topical cream, is effective and safe at treating facial angiofibroma in patients 6 years of age and over.

Phase 1

Conditions: Facial Angiofibromas Associated with Tuberous Sclerosis Complex
MedDRA version: 20.0Level: PTClassification code 10002429Term: AngiofibromaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.0Level: PTClassification code 10073678Term: Juvenile angiofibromaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Registration Number: EUCTR2019-000752-34-SK

Lead Sponsor: DSLP

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 120

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
Are the trial subjects under 18? yes
Number of subjects for this age range: 90
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

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

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The Primary Efficacy Objective is to determine and compare the efficacy of two different strengths of Rapamycin cream, topical and placebo over 26 weeks of once-daily treatment for Facial Angiofibromas associated with Tuberous Sclerosis Complex (TSC).;Secondary Objective: The Safety Objective of the study is to determine and compare the incidence of treatment-emergent adverse events (TEAEs) over the course of the study among the three treatment groups.;Primary end point(s): The percentage of patients obtaining successful treatment based on a blind assessment using the IGA scale after 26 weeks treatment or at last visit if early withdrawal/discontinuation;Timepoint(s) of evaluation of this end point: After 26 weeks treatment

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Time to treatment success<br>- Change from baseline in IGA after 26 weeks treatment or at last visit if early withdrawal/discontinuation<br>- Change from baseline in FASI after 26 weeks treatment or at last visit if early withdrawal/discontinuation<br>- Subjective (patient or parent/caregiver) improvement rating from first visit (V0) after 26 weeks treatment or at last visit if early withdrawal/discontinuation<br>- Objective (clinician) improvement rating from first visit (V0) after 26 weeks treatment or at last visit if early withdrawal/discontinuation<br>- Categorical Improvement of Facial Angiofibroma from first visit (V0) after 26 weeks treatment;Timepoint(s) of evaluation of this end point: After 2, 8, 14, 20 and 26 weeks of treatment

Related Trials