Neurofibromatosis type 1
Conditions
Interventions
0.2% NPC-12G gel topically administered twice a day for 52 weeks
0.4% NPC-12G gel topically administered twice a day for 52 weeks
Placebo gel topically administered twice a day for 52 weeks
Sponsors
Osaka University Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Patients diagnosed as neurofibromatosis type 1 according to the clinical diagnostic criteria in guideline of Japanese Dermatological Association 2) Patients with 10 skin lesions (at least 5 lesions) that can be selected from those of the maximum size. The skin lesions can be taken pictures by 3D camera and must satisfy all of the following conditions. a)Tumors of 3 mm or longer in longest diameter measured by ruler. b)Tumors located solitary c)Skin lesions without hair d)Tumors of mountain-like shape with no shadows when taken pictures from above (bulb shape or gourd shape tumor should not be selected) e)Tumors not located on the skin with large expansion and contraction such as inside of the elbow or the scruff. 3) Patients who is 3 years old or elder 4) Patients who provide written informed consent by themselves or legally acceptable representatives
Exclusion criteria
Exclusion criteria: 1) Patients who treated with mTOR inhibitors (sirolimus, everolimus or temsirolimus), within 12 months prior to enrollment. 2) Patients who treated with RAS-MAPK inhibitors (sorafenib, regorafenib or lenvatinib), within 12 months prior to enrollment. 3) Patients who have active infectious lesions. 4) Patients who have abnormal findings (pneumonic lesions) by chest X-ray inspection. 5) Patients with creatinine clearance less than 50ml/min. 6) Patients with uncontrolled dyslipidemia (serum triglyceride is 500mg / dL or more, or LDL cholesterol is 190mg / dL or more even treated) 7) Patients who have severe complications such as cardiac disease, liver disease, pulmonary disease, hematological disorder or malignant tumor . 8) Patients who have previously experienced alcoholic sensitivity or allergy to sirolimus. 9) Patients who are pregnant or lactating. 10) Patients who cannot agree to use effective contraceptive methods during the study period and until 8 weeks after treatment. 11) Patients who have entered another clinical trial within 6 months prior to enrollment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Response rate on the changes from baseline in the volume of cutaneous tumor (measured with 3D camera) after 52 week treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) Time course of changes from baseline in the volume of cutaneous tumor (measured with 3D camera) 2) Response rate on the changes from baseline in the volume of cutaneous tumor (measured with 3D camera) after 16 week, 28 week and 40 week treatment 3) Time course of changes from baseline in the area of cutaneous tumor (measured with ruler) 4) Improvement of cutaneous lesions after 28 and 52 week treatment judged by physician in charge | — |
Countries
Japan
Contacts
Public ContactMari Kaneda
Osaka University Hospital Department of Dermatology
Outcome results
None listed