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

NCT ID: NCT03826628

Last Updated: 2023-09-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 107 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-07-28
• Study Completion Date: 2022-09-01

Brief 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).

Detailed 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).

Conditions

Facial Angiofibroma; Tuberous Sclerosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

0.5% Rapamycin cream, topical

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

Group Type: EXPERIMENTAL

rapamycin

Intervention Type: DRUG

Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks

1.0% Rapamycin cream, topical

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

Group Type: EXPERIMENTAL

rapamycin

Intervention Type: DRUG

Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks

Placebo

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

Group Type: PLACEBO_COMPARATOR

placebo

Intervention Type: DRUG

Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks

Interventions

rapamycin

Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks

Intervention Type: DRUG

placebo

Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks

Intervention Type: DRUG

Other Intervention Names

sirolimus

Eligibility 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

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Dermatology Specialties Limited Partnership

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ioana Stanescu

Role: STUDY_DIRECTOR

Dermatology Specialties Limited Partnership

Locations

Phoenix Children's Hospital

Phoenix, Arizona, United States

University of California San Diego

La Jolla, California, United States

All Children's Research Institute

St. Petersburg, Florida, United States

Spectrum Health

Grand Rapids, Michigan, United States

Mayo Clinic

Rochester, Minnesota, United States

University of Virginia

Charlottesville, Virginia, United States

Children's Health Queensland

Brisbane, Queensland, Australia

Fakultni nemocnice Brno

Brno, , Czechia

Bethesda Children's Hospital of the Hungarian Reformed Church

Budapest, , Hungary

University of Pécs

Pécs, , Hungary

Canterbury District Health Board

Christchurch, Canterbury, New Zealand

Clinic of Neurology and Psychiatry for Children and Youth

Belgrade, , Serbia

Clinical Center of Serbia

Belgrade, , Serbia

Narodný ústav detských chorȏb

Bratislava, , Slovakia

Clinica Universidad de Navarra

Pamplona, Navarre, Spain

Clínica Universidad de Navarra

Madrid, , Spain

National Taiwan University Hospital

Taipei, , Taiwan

Countries

United States; Australia; Czechia; Hungary; New Zealand; Serbia; Slovakia; Spain; Taiwan

References

Aitken P, Stanescu I, Boddington L, Mahon C, Fogarasi A, Liao YH, Ivars M, Moreno-Artero E, Trauner D, DeRoos ST, Jancic J, Nikolic M, Balazova P, Price HN, Hadzsiev K, Riney K, Stapleton S, Tollefson MM, Bauer D, Pinkova B, Atkinson H. A novel rapamycin cream formulation improves facial angiofibromas associated with tuberous sclerosis complex: a double-blind randomized placebo-controlled trial. Br J Dermatol. 2023 Oct 25;189(5):520-530. doi: 10.1093/bjd/ljad243.

Reference Type: RESULT

PMID: 37463422 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

DSLP-01

Identifier Type: -

Identifier Source: org_study_id