Unique ID issued by UMIN | UMIN000036527 |
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Receipt number | R000041614 |
Scientific Title | 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 |
Date of disclosure of the study information | 2019/04/22 |
Last modified on | 2021/10/19 10:35:15 |
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 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
Japan |
Neurofibromatosis type 1
Dermatology |
Others
NO
To evaluate efficacy and safety of NPC-12G at doses of 0.2% and 0.4% compared with placebo based on the changes in the volume of cutaneous tumor in patients with Neurofibromatosis type 1
Safety,Efficacy
Exploratory
Phase II,III
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
Interventional
Parallel
Randomized
Individual
Double blind -all involved are blinded
Placebo
NO
NO
Institution is not considered as adjustment factor.
NO
No need to know
3
Treatment
Medicine |
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
3 | years-old | <= |
Not applicable |
Male and Female
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.
63
1st name | Mari |
Middle name | |
Last name | Kaneda |
Osaka University Hospital
Department of Dermatology
565-0871
2-15 Yamadaoka, Suita, Osaka, Japan
06-6879-3031
mkaneda@derma.med.osaka-u.ac.jp
1st name | Mari |
Middle name | |
Last name | Kaneda |
Osaka University Hospital
Department of Dermatology
565-0871
2-15 Yamadaoka, Suita, Osaka, Japan
06-6879-3031
mkaneda@derma.med.osaka-u.ac.jp
Osaka University Hospital
Japan Agency for Medical Research and Development
Japanese Governmental office
Institutional Review Board of Osaka University hospital
2-2 Yamadaoka, Suita, Osaka, Japan
06-6210-8290
shiken@hp-crc.med.osaka-u.ac.jp
NO
大阪大学医学部附属病院(大阪府)、東京慈恵会医科大学附属病院(東京都)、
鳥取大学医学部附属病院(鳥取県)、福岡大学病院(福岡県)
2019 | Year | 04 | Month | 22 | Day |
Unpublished
76
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.
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 to the skin lesion of the patients with neurofibromatosis type 1 for 52 weeks.
2021 | Year | 07 | Month | 05 | Day |
Male: 22 cases, Female: 54 cases
Mean age: 48.3,
Minimum age: 19, Maximum age: 78
Informed consent obtained: 84 cases
Enrolled: 76 cases
0.2% group: 25 cases, 0.4% group: 24 cases, Placebo group: 27 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. 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. 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.
Completed
2019 | Year | 03 | Month | 22 | Day |
2019 | Year | 03 | Month | 22 | Day |
2019 | Year | 05 | Month | 10 | Day |
2021 | Year | 02 | Month | 28 | Day |
2021 | Year | 03 | Month | 31 | Day |
2021 | Year | 03 | Month | 31 | Day |
2019 | Year | 04 | Month | 16 | Day |
2021 | Year | 10 | Month | 19 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000041614
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