UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000015081
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

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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 or person

Institute

Department of Dermatology
Graduate School of Medicine, Osaka University

Institute

Department

Personal name



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

Value
https://center6.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