UMIN-CTR Clinical Trial

Public title

Clinical trial for development of topical rapamycin treatment for white macules due to tuberous sclerosis complex, vitiligo vulgaris, and other congenital diseases

Acronym

Clinical trial for development of topical rapamycin treatment for white macules due to tuberous sclerosis complex, vitiligo vulgaris, and other congenital diseases

Scientific Title

Clinical trial for development of topical rapamycin treatment for white macules due to tuberous sclerosis complex, vitiligo vulgaris, and other congenital diseases

Scientific Title:Acronym

Clinical trial for development of topical rapamycin treatment for white macules due to tuberous sclerosis complex, vitiligo vulgaris, and other congenital diseases

Region

Japan

Condition

tuberous sclerosis complex, vitiligo vulgaris, white macules due to congenital diseases

Classification by specialty

Dermatology

Classification by malignancy

Others

Genomic information

NO

Narrative objectives1

The purpose of this trial is to develop an effective and safe treatment for white macules due to tuberous sclerosis complex, vitiligo vulgaris, and other congenital diseases.

Basic objectives2

Safety,Efficacy

Basic objectives -Others

Trial characteristics_1

Trial characteristics_2

Pragmatic

Developmental phase

Phase II,III

Primary outcomes

Changes in color and size of white macules at 4, 8, and 12 weeks

Key secondary outcomes

Appearance of contact dermatitis
Rapamycin levels in whole blood
Numbers and shapes of melanocyte in specimens of skin tissue in the cases who agree with skin biopsy

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 (base is carboxyvinyl polymer) on white macules of the face twice a day for 12 weeks.
When it is possible, application of 0.2% rapamycin gel twice a day for 12 weeks on white macules of the non-exposed part to the sun.

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

Not applicable

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, or patients diagnosed clinically as vitiligo vulgaris, piebaldism, hypomelanosis of Ito, or nevus depigmentosus.
2) Patients who want treatment for white macules.
3) Patients who cannot receive other effective treatments.
4) Patients who would not want to receive other treatments because of social or cosmetic reasons.

Key exclusion criteria

1) Patients with severe mental retardation who cannot carry out this treatment plan.
2) Patients who have allergy to macrolide antibiotics.
3) Patients who received oral administration of rapamycin or RAD001 within six months prior to the study entry.
4) Patients who received topical treatment of tacrolimus, vitamin D, or steroid within three months prior to the study entry.
5) Patients who received phototherapy or surgical therapy within six months prior to the study entry.
6) Patients during pregnancy or lactation
7) Patients who were judged unsuitable for this study by the investigator.

Target sample size

21

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

Japan

Co-sponsor

Name of secondary funder(s)

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

Date of disclosure of the study information

2011

Year

08

Month

03

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

Enrolling by invitation

Date of protocol fixation

2011

Year

07

Month

25

Day

Date of IRB

Anticipated trial start date

2011

Year

08

Month

01

Day

Last follow-up date

2015

Year

03

Month

31

Day

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Registered date

2011

Year

08

Month

03

Day

Last modified on

2013

Year

09

Month

12

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000007206