Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complex

• Register: JPRN
• Last refreshed on: 17 October 2023
• Main ID: JPRN-UMIN000015114
• Date of registration: 10/09/2014
• Prospective Registration: Yes
• Primary sponsor: Department of Dermatology Graduate School of Medicine, Osaka University
• Public title: Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complex
• Scientific title: Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complex - Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complex
• Date of first enrolment: 2014/09/11
• Target sample size: 24
• Recruitment status: Recruiting
• URL: https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000017546
• Study type: Interventional
• Study design: Single arm Non-randomized
• Phase: Phase II,III

Countries of recruitment

Japan

Contacts

• Name: Mari Wataya-Kaneda
• Affiliation: Graduate School of Medicine, Osaka University Department of Dermatology

• Name: Mari Wataya-Kaneda
• Affiliation: Graduate School of Medicine, Osaka University Department of Dermatology

Key inclusion & exclusion criteria

Inclusion criteria:
Exclusion criteria: 1) Patients who cannot carry out this treatment plan. 2) Patients who have a severe erosion or ulcer on the lesion. 3) Patients who have allergy to macrolide antibiotics. 4) Patients who received oral administration of rapamycin (sirolimus) , everolimus or temsirolimus within 12 months prior to the study entry. 5) Patients who received topical treatment of tacrolimus within three months prior to the study entry. 6) Patients who received laser or surgical therapy to the target lesion within six months prior to the study entry. 7) Patients who have complications such as severe heart disease, liver disease, lung disease, or blood disorders, and are considered unsuitable to participate in this study. 8) Patients with alcohol intolerance. 9) Patients who are pregnant or lactating. 10) Patients who cannot agree to use effective methods of contraception during this study. 11) Patients who were judged unsuitable for this study by the investigator.

Age minimum: Not applicable
Age maximum: 70years-old
Gender: Male and Female

Health Condition(s) or Problem(s) studied

Tuberous sclerosis complex

Intervention(s)

Application of 0.2% rapamycin gel on skin lesions of the face and trunk twice a day for 12 weeks.

Primary Outcome(s)

Improvements in redness, papule size and flatness of facial angiofibroma Adverse events

Secondary Outcome(s)

Improvements in erythema, plaque, and white macules of the face Improvements in shagreen patch and white macules of the trunk The comprehensive improvement judged by doctor Patient satisfaction Blood level of rapamycin Results of blood test and urinalysis

Source(s) of Monetary Support

Ministry of Education, Culture, Sports, Science and Technology of Japan

Ministry of Health, Labour and Welfare

Ethics review

Status: YES
Approval date:
Contact:

Results

• Results available: Yes
• Date Completed: 31/12/2015