UMIN-CTR Clinical Trial

Public title

Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complex

Acronym

Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complex

Scientific Title

Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complex

Scientific Title:Acronym

Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complex

Region

Japan

Condition

Tuberous sclerosis complex

Classification by specialty

Dermatology

Classification by malignancy

Others

Genomic information

NO

Narrative objectives1

The purpose of this study is to explore the safety and the efficacy of topical rapamycin gel for skin lesions in tuberous sclerosis complex.

Basic objectives2

Safety,Efficacy

Basic objectives -Others

Trial characteristics_1

Confirmatory

Trial characteristics_2

Developmental phase

Phase II,III

Primary outcomes

Improvements in redness, papule size and flatness of facial angiofibroma
Adverse events

Key secondary outcomes

Improvements in erythema, plaque, and white macules of the face
Improvements in shagreen patch and white macules of the trunk
The comprehensive improvement judged by doctor
Patient satisfaction
Blood level of rapamycin
Results of blood test and urinalysis

Study type

Interventional

Basic design

Single arm

Randomization

Non-randomized

Randomization unit

Blinding

Open -no one is blinded

Control

Uncontrolled

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

No. of arms

1

Purpose of intervention

Treatment

Type of intervention

Medicine

Interventions/Control_1

Application of 0.2% rapamycin gel on skin lesions of the face and trunk twice a day for 12 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

Age-lower limit

Not applicable

Age-upper limit

70

years-old

>

Gender

Male and Female

Key inclusion criteria

1) Patients diagnosed as tuberous sclerosis complex based on the clinical diagnostic criteria in guideline of Japanese Dermatological Association.
2) Patients who have solitary angiofibromas on the face.
3) Patients who are not suitable for laser or surgical treatments, or who don't want surgical treatments.
4) Patients who or whose guardian can apply medicine as planed according to the instructions of the doctor
5) Patients who or whose guardian can give the written consent by oneself in understanding after having received enough explanation on this study.
6) Patients under 70 years old

Key exclusion criteria

1) Patients who cannot carry out this treatment plan.
2) Patients who have a severe erosion or ulcer on the lesion.
3) Patients who have allergy to macrolide antibiotics.
4) Patients who received oral administration of rapamycin (sirolimus) , everolimus or temsirolimus within 12 months prior to the study entry.
5) Patients who received topical treatment of tacrolimus within three months prior to the study entry.
6) Patients who received laser or surgical therapy to the target lesion within six months prior to the study entry.
7) Patients who have complications such as severe heart disease, liver disease, lung disease, or blood disorders, and are considered unsuitable to participate in this study.
8) Patients with alcohol intolerance.
9) Patients who are pregnant or lactating.
10) Patients who cannot agree to use effective methods of contraception during this study.
11) Patients who were judged unsuitable for this study by the investigator.

Target sample size

24

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

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

Institute

Department of Dermatology
Graduate School of Medicine, Osaka University

Institute

Department

Personal name

Organization

Ministry of Health, Labour and Welfare

Organization

Division

Category of Funding Organization

Nationality of Funding Organization

Co-sponsor

Japan

Name of secondary funder(s)

Ministry of Education, Culture, Sports, Science and Technology of Japan

Organization

Address

Tel

Email

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

大阪大学医学部附属病院（大阪府）

Date of disclosure of the study information

2014

Year

09

Month

10

Day

URL releasing protocol

Publication of results

Unpublished

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

Recruitment status

Open public recruiting

Date of protocol fixation

2014

Year

07

Month

28

Day

Date of IRB

Anticipated trial start date

2014

Year

09

Month

11

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 related information

Registered date

2014

Year

09

Month

10

Day

Last modified on

2014

Year

09

Month

10

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000017546