neurofibromatosis type1
Conditions
Interventions
1) Application of 0.2% rapamycin gel on one third of the tumor once a day for 2 weeks. 2) Check the skin findings, blood test results, blood level of rapamycin and adverse events after 2 weeks of tr
Sponsors
Department of Dermatology Graduate School of Medicine, Osaka University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Patients diagnosed as neurofibromatosis type1 based on the clinical diagnostic criteria in guideline of Japanese Dermatological Association. 2) Patients who have a disabling large diffuse plexiform neurofibroma and cannot receive other effective treatments 3) Patients who can give the written consent by oneself in understanding after having received enough explanation on this examination
Exclusion criteria
Exclusion criteria: 1) Patients who cannot carry out this treatment plan. 2) Patients who have allergy to macrolide antibiotics. 3) Patients who received oral or topical administration of rapamycin or RAD001 within six months prior to the study entry. 4) Patients who received topical treatment of tacrolimus within three months prior to the study entry. 5) Patients who received surgical therapy within six months prior to the study entry. 6) Patients who received radiotherapy or Neutron Capture Therapy within six months prior to the study entry. 7) Patients during pregnancy or lactation 8) Patients who were judged unsuitable for this study by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Skin findings at the site of topical application Blood test results Blood level of rapamycin Serious adverse events Changes in size of tumor after 12 weeks of treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Changes in contrast-enhanced MRI data Histological findings in biopsy specimens of the tumor | — |
Countries
Japan
Contacts
Public ContactMari Wataya-Kaneda
Graduate School of Medicine, Osaka University Department of Dermatology
Outcome results
None listed