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A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of NPC-12G for Skin Lesions in Patients with Neurofibromatosis type 1 (NEDOC-2 Study)

A Double-Blind Study to Evaluate Efficacy and Safety of NPC-12G in Patients with Neurofibromatosis type 1 (NEDOC-2 Study)

76

Male: 22 cases, Female: 54 cases Mean age: 48.3, Minimum age: 19, Maximum age: 78 0.2% group: 25 cases, 0.4% group: 24 cases, Placebo group: 27 cases

Informed consent obtained: 84 cases Enrolled: 76 cases Safety evaluated: 76 cases Efficacy evaluated: 75 cases

Adverse events observed were at 92.0% (23/25) of the cases in the 0.2% group, 87.5% (21/24) in the 0.4% group and 70.4% (19/27) in the placebo group. No death was observed. Side effects observed were at 52.0% (13/25) of the cases in the 0.2% group, 45.8% (11/24) in the 0.4% group and 37.0% (10/27) in the placebo group. Side effects frequently observed were pruritus and dry skin in both of NPC12-G group and placebo group. No serious side effects were observed.

Primary outcome measures showed there were no responders in any of the groups with the evaluation of the response rate after 52 weeks administration and there were no statistical differences observed between the NPC12-G group and the placebo group. Regarding the secondary outcome measures, there were no statistical differences observed between the NPC12-G group and the placebo group.

0.2% NPC-12G, 0.4% NPC12-G and placebo were topically administered to the skin lesion of the patients with neurofibromatosis type 1 for 52 weeks. There were no statistical differences between the NPC12-G group and the placebo group regarding the efficacy evaluation and no tumor shrinkage effects of NPC12-G were observed. There were no serious side effects observed and there were no special concerns regarding the safety of 0.2% NPC-12G and 0.4% NPC12-G when topically administered for 52 weeks.

Aug. 31, 2021

No

https://jrct.mhlw.go.jp/latest-detail/jRCT2051190008

Kaneda Mari

Osaka University Hospital

2-15 Yamadaoka Suita-city Osaka, Japan

mkaneda@derma.med.osaka-u.ac.jp

Kanda Mari

Osaka University Hospital

2-15 Yamadaoka Suita-city Osaka, Japan

+81-6-6879-3031

mkaneda@derma.med.osaka-u.ac.jp

Complete

May. 10, 2019

Interventional

randomized controlled trial

double blind

placebo control

parallel assignment

treatment purpose

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

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.

Both

Neurofibromatosis type 1

0.2% NPC-12G gel, 0.4% NPC-12G gel or placebo gel is topically administered twice a day for 52 weeks

Neurofibromatosis type 1

Topical administration

D009456

D000287

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

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

+81-6-6210-8290

Mar. 22, 2019

none