Unique ID issued by UMIN | UMIN000021030 |
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Receipt number | R000024265 |
Scientific Title | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1 |
Date of disclosure of the study information | 2016/02/22 |
Last modified on | 2017/05/02 14:56:03 |
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1
Japan |
Neurofibromatosis type 1
Dermatology |
Others
NO
To evaluate safety and efficacy of OSD-001 at doses of 0.2% and 0.4% based on the changes in the size of cutaneous tumor in patients with Neurofibromatosis type 1
Safety,Efficacy
Exploratory
Explanatory
Phase II
Changes from baseline in the size of cutaneous tumor (CT scan) after 24 week treatment
1. Changes from baseline in the size of cutaneous tumor (measured by ruler) after 24 week treatment
2. Improvements of cutaneous lesions after 12 or 24 week treatment
3. Histopathological evaluation of cutaneous tumor after 24 week treatment
4. Evaluation of Dermatology Life Quality Index (DLQI) after 12 or 24 week treatment
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% Sirolimus gel twice daily 24 weeks topical application
0.4% Sirolimus gel twice daily 24 weeks topical application
Placebo gel twice daily 24 weeks topical application
16 | years-old | <= |
70 | years-old | > |
Male and Female
1) Patients diagnosed as neurofibromatosis type 1 based on the clinical diagnostic criteria in guideline of Japanese Dermatological Association
2) Patients who have the skin lesions of 450 cm2 or less to be evaluable
3) Patients whose skin lesions increase within 6 months .
4) Patients who is 16 years old or elder and younger than 70 years old
1) Patients who received radiotherapy or neutron capture therapy on skin lesions within 12 months prior to enrollment.
2) Patients who have received surgical therapy on skin lesions within 6 months prior to enrollment.
3) Patients who treated with sirolimus, everolimus or temsirolimus (mTOR inhibitors), within 6 months prior to enrollment.
4) Patients who received topical treatment of tacrolimus within 3 months prior to enrollment.
5) Patients who have severe complications such as cardiac disease, liver disease, pulmonary disease, hematological disorder .
6) Patients who have previously experienced alcoholic sensitivity or allergy to sirolimus.
7) Patients who are pregnant or lactating.
8) Patients who have entered another clinical trial within 6 months prior to enrollment.
18
1st name | |
Middle name | |
Last name | Mari Wataya-Kaneda |
Graduate School of Medicine, Osaka University
Department of Dermatology
2-2, Yamadaoka, Suita, Osaka 565-0871, Japan
06-6879-5111
mkaneda@derma.med.osaka-u.ac.jp
1st name | |
Middle name | |
Last name | Mari Wataya-Kaneda |
Graduate School of Medicine, Osaka University
Department of Dermatology
2-2, Yamadaoka, Suita, Osaka 565-0871, Japan
06-6879-5111
mkaneda@derma.med.osaka-u.ac.jp
Osaka University Hospital
Japan Agency for Medical Research and Development
Other
Japan
NO
大阪大学医学部附属病院(大阪府)
2016 | Year | 02 | Month | 22 | Day |
Unpublished
Completed
2016 | Year | 01 | Month | 05 | Day |
2016 | Year | 03 | Month | 16 | Day |
2017 | Year | 01 | Month | 20 | Day |
2017 | Year | 02 | Month | 28 | Day |
2017 | Year | 03 | Month | 31 | Day |
2017 | Year | 04 | Month | 30 | Day |
2016 | Year | 02 | Month | 15 | Day |
2017 | Year | 05 | Month | 02 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000024265
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