Unique ID issued by UMIN | UMIN000015081 |
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Receipt number | R000017188 |
Scientific Title | Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1 |
Date of disclosure of the study information | 2014/09/07 |
Last modified on | 2014/09/07 21:18:47 |
Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1
Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1
Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1
Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1
Japan |
neurofibromatosis type1
Dermatology |
Others
NO
The purpose of this study is to explore the safety and the effectiveness of rapamycin topical medication for large diffuse plexiform neurofibroma of neurofibromatosis type 1
Safety,Efficacy
Exploratory
Phase II,III
Skin findings at the site of topical application
Blood test results
Blood level of rapamycin
Serious adverse events
Changes in size of tumor after 12 weeks of treatment
Changes in contrast-enhanced MRI data
Histological findings in biopsy specimens of the tumor
Interventional
Single arm
Non-randomized
Open -no one is blinded
Uncontrolled
1
Treatment
Medicine |
1) Application of 0.2% rapamycin gel on one third of the tumor once a day for 2 weeks.
2) Check the skin findings, blood test results, blood level of rapamycin and adverse events after 2 weeks of treatment.
If there is no problem, perform application of 0.2% rapamycin gel on two-thirds of the tumor once a day for another 2 weeks.
3) Check the skin findings, blood test results, blood level of rapamycin and adverse events after a total of 4 weeks of treatment.
If there is no problem, perform application of 0.2% rapamycin gel on the whole surface of the tumor once a day for another 8 weeks.
20 | years-old | <= |
Not applicable |
Male and Female
1) Patients diagnosed as neurofibromatosis type1 based on the clinical diagnostic criteria in guideline of Japanese Dermatological Association.
2) Patients who have a disabling large diffuse plexiform neurofibroma and cannot receive other effective treatments
3) Patients who can give the written consent by oneself in understanding after having received enough explanation on this examination
1) Patients who cannot carry out this treatment plan.
2) Patients who have allergy to macrolide antibiotics.
3) Patients who received oral or topical administration of rapamycin or RAD001 within six months prior to the study entry.
4) Patients who received topical treatment of tacrolimus within three months prior to the study entry.
5) Patients who received surgical therapy within six months prior to the study entry.
6) Patients who received radiotherapy or Neutron Capture Therapy within six months prior to the study entry.
7) Patients during pregnancy or lactation
8) Patients who were judged unsuitable for this study by the investigator.
3
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
06-6879-3031
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
06-6879-3031
mkaneda@derma.med.osaka-u.ac.jp
Department of Dermatology
Graduate School of Medicine, Osaka University
Ministry of Health, Labour and Welfare
Japan
Ministry of Education, Culture, Sports, Science and Technology of Japan
NO
大阪大学医学部附属病院
2014 | Year | 09 | Month | 07 | Day |
Unpublished
Enrolling by invitation
2014 | Year | 07 | Month | 28 | Day |
2014 | Year | 09 | Month | 08 | Day |
2015 | Year | 12 | Month | 31 | Day |
2014 | Year | 09 | Month | 07 | Day |
2014 | Year | 09 | Month | 07 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000017188
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