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Name:
UMIN ID:

Recruitment status Enrolling by invitation
Unique ID issued by UMIN UMIN000015081
Receipt No. 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

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Basic information
Public title Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1
Acronym Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1
Scientific Title Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1
Scientific Title:Acronym Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1
Region
Japan

Condition
Condition neurofibromatosis type1
Classification by specialty
Dermatology
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 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
Basic objectives2 Safety,Efficacy
Basic objectives -Others
Trial characteristics_1 Exploratory
Trial characteristics_2
Developmental phase Phase II,III

Assessment
Primary outcomes 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
Key secondary outcomes Changes in contrast-enhanced MRI data
Histological findings in biopsy specimens of the tumor

Base
Study type Interventional

Study design
Basic design Single arm
Randomization Non-randomized
Randomization unit
Blinding Open -no one is blinded
Control Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms 1
Purpose of intervention Treatment
Type of intervention
Medicine
Interventions/Control_1 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.
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit
20 years-old <=
Age-upper limit

Not applicable
Gender Male and Female
Key inclusion criteria 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
Key exclusion criteria 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.
Target sample size 3

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Mari Wataya-Kaneda
Organization Graduate School of Medicine, Osaka University
Division name Department of Dermatology
Zip code
Address 2-2, Yamadaoka, Suita, Osaka 565-0871, JAPAN
TEL 06-6879-3031
Email mkaneda@derma.med.osaka-u.ac.jp

Public contact
Name of contact person
1st name
Middle name
Last name Mari Wataya-Kaneda
Organization Graduate School of Medicine, Osaka University
Division name Department of Dermatology
Zip code
Address 2-2, Yamadaoka, Suita, Osaka 565-0871, JAPAN
TEL 06-6879-3031
Homepage URL
Email mkaneda@derma.med.osaka-u.ac.jp

Sponsor
Institute Department of Dermatology
Graduate School of Medicine, Osaka University
Institute
Department

Funding Source
Organization Ministry of Health, Labour and Welfare
Organization
Division
Category of Funding Organization
Nationality of Funding Organization Japan

Other related organizations
Co-sponsor
Name of secondary funder(s) Ministry of Education, Culture, Sports, Science and Technology of Japan

IRB Contact (For public release)
Organization
Address
Tel
Email

Secondary IDs
Secondary IDs NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions 大阪大学医学部附属病院

Other administrative information
Date of disclosure of the study information
2014 Year 09 Month 07 Day

Related information
URL releasing protocol
Publication of results Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status Enrolling by invitation
Date of protocol fixation
2014 Year 07 Month 28 Day
Date of IRB
Anticipated trial start date
2014 Year 09 Month 08 Day
Last follow-up date
2015 Year 12 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2014 Year 09 Month 07 Day
Last modified on
2014 Year 09 Month 07 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000017188

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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