A Double-Blind Study to Evaluate Efficacy and Safety of NPC-12G in Patients with Neurofibromatosis type 1

JRCT ID: jRCT2051190008

Registered date:22/04/2019

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Neurofibromatosis type 1
• Date of first enrollment: 22/05/2019
• Target sample size: 63
• Study type: Interventional
• Intervention(s): 0.2% NPC-12G gel, 0.4% NPC-12G gel or placebo gel is topically administered twice a day for 52 weeks

Outcome(s)

• Primary Outcome: Response rate on the changes from baseline in the volume of cutaneous tumor (measured with 3D camera) after 52 week treatment

Secondary Outcome

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

Key inclusion & exclusion criteria

• Age minimum: >= 3age old
• Age maximum: Not applicable
• Gender: Both

Include 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

Exclude 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.

Related Information

• Primary Sponsor: Kaneda Mari