Tuberous sclerosis complex
Conditions
Interventions
Application of 0.2% rapamycin gel on skin lesions of the face and trunk twice a day for 12 weeks.
Sponsors
Department of Dermatology Graduate School of Medicine, Osaka University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Patients diagnosed as tuberous sclerosis complex based on the clinical diagnostic criteria in guideline of Japanese Dermatological Association. 2) Patients who have solitary angiofibromas on the face. 3) Patients who are not suitable for laser or surgical treatments, or who don't want surgical treatments. 4) Patients who or whose guardian can apply medicine as planed according to the instructions of the doctor 5) Patients who or whose guardian can give the written consent by oneself in understanding after having received enough explanation on this study. 6) Patients under 70 years old
Exclusion 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Improvements in redness, papule size and flatness of facial angiofibroma Adverse events | — |
Secondary
| Measure | Time frame |
|---|---|
| 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 | — |
Countries
Japan
Contacts
Public ContactMari Wataya-Kaneda
Graduate School of Medicine, Osaka University Department of Dermatology
Outcome results
None listed