UMIN-CTR Clinical Trial

Public title

Clinical study of combination local injection sclerotherapy of bleomycin and OK-432 for intractable lymphatic malformations

Acronym

Study of combination sclerotherapy of bleomycin and OK-432 for LMs

Scientific Title

Clinical study of combination local injection sclerotherapy of bleomycin and OK-432 for intractable lymphatic malformations

Scientific Title:Acronym

Study of combination sclerotherapy of bleomycin and OK-432 for LMs

Region

Japan

Condition

Lymphangioma and other lymphatic diseases with similar lesion

Classification by specialty

Surgery in general	Vascular surgery	Pediatrics
Ophthalmology	Dermatology	Oto-rhino-laryngology
Oral surgery	Plastic surgery	Aesthetic surgery
Operative medicine

Classification by malignancy

Others

Genomic information

NO

Narrative objectives1

To examine efficacy and safety of combination local injection sclerotherapy of bleomycin and OK-432

Basic objectives2

Safety,Efficacy

Basic objectives -Others

Trial characteristics_1

Confirmatory

Trial characteristics_2

Pragmatic

Developmental phase

Phase III

Primary outcomes

The proportion of subjects that the volume of therapeutic target area in the lesion is reduced 30% or more

Key secondary outcomes

Appearance, degree of uplift due to lesions, clinical symptoms such as lymph leakage and bleeding, pain, and frequency of fever / redness,the subjects' impression of their improvement and their degree of satisfaction. Changes in the lung shadows on chest X-ray.

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

"OK-432 and bleomycin slolution" (0.05 kE / mL and 0.5 mg / mL, respectively) will be injected into the lesion with water-soluble contrast agent.
Maximum dose of bleomycin is 10 mg / dose and 5 mg/kgBW.
Cumulative maximum dose in repeated treatment is 10 mg/kgBW.

OK-432 concentration might be redued to the range from 0.01 to 0.05 kE / mL by the attending physician's judgment, for reasons such as reducing the inflammation induced by OK-432.

[Exit criteria]
When subjects and family members reach satisfaction with the level of reduction of the lesion.
When the cumulative dose reaches the maximum dose.

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

Patients requiring treatment in the National Center for Child Health and Development, diagnosed as lymphangioma (lymphatic malformation, common and cystic lymphatic malformation) or other lymphatic disease with similar pathological tissue.

Among the above-mentioned subject, patients satisfying the following conditions A and B, and having understood and agreed to the research by document, will be included.
Condition A; satisfying the following condition a or b.
a; spongy lesions remaining after the OK-432 therapy in the past
b; spongy lesion untreated with sclerotherapy and expected of no efficacy of OK-432
Condition B; surgical resection is difficult to apply.

Key exclusion criteria

Patients with any of the following diseases will be excluded.
Severe lung dysfunction, lung fibrosis lesions and significant lung lesions, history of hypersensitivity to bleomycin hydrochloride and similar compounds to the (peplomycin), severe renal dysfunction, severe heart disease, and history of radiation to the chest and the periphery

Target sample size

21

Name of lead principal investigator

1st name	Akihiro
Middle name
Last name	Fujino

Organization

National Center for Child Health and Development

Division name

Division of Pediatric Surgery

Zip code

157-8535

Address

2-10-1 Okura, Setagaya-Ku, Tokyo 157-8535

TEL

03-3416-0181

Email

fujino-a@ncchd.go.jp

Name of contact person

1st name	Akihiro
Middle name
Last name	Fujino

Organization

National Center for Child Health and Development

Division name

Division of Pediatric Surgery

Zip code

157-8535

Address

2-10-1 Okura, Setagaya-Ku, Tokyo 157-8535

TEL

03-3416-0181

Homepage URL

Email

fujino-a@ncchd.go.jp

Institute

National Center for Child Health and Development

Institute

Department

Personal name

Organization

Japan Agency for Medical Research and Development

Organization

Division

Category of Funding Organization

Japanese Governmental office

Nationality of Funding Organization

Japan

Co-sponsor

Name of secondary funder(s)

Organization

Certified Review Board of National Center for Child Health and Development

Address

2-10-1 Okura, Setagaya-ku, Tokyo, Tokyo

Tel

03-3416-0181

Email

rinken@ncchd.go.jp

Secondary IDs

YES

Study ID_1

jRCTs031180265

Org. issuing International ID_1

jRCT

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

国立成育医療研究センター（東京都）

Date of disclosure of the study information

2016

Year

08

Month

05

Day

URL releasing protocol

Publication of results

Unpublished

URL related to results and publications

Number of participants that the trial has enrolled

29

Results

Results date posted

Results Delayed

Delay expected

Results Delay Reason

The principal investigator's work on the paper has been delayed.

Date of the first journal publication of results

Baseline Characteristics

　The planned subjects were "patients seen at the National Center for Child Health and Development with a diagnosis of lymphangioma (lymphangitic malformation, general or cystic type lymphangitic malformation) and diseases with similar tissue requiring treatment", regardless of age or gender.
　Patients meeting the following criteria for refractory disease were selected as selection criteria.
Among the above-mentioned subjects, patients who met the following conditions A and B and who understood and agreed to the written explanation.
A, The patient meets either (1) or (2) below.
(1) Patients with residual spongiform lesions after previous treatment with OK-432.
(ii) Sclerotherapy untreated, but the lesion is spongiform and likely to be ineffective for OK-432.
B, Difficulty in choosing surgical resection.

　According to the above conditions, 12 male and 17 female patients between the ages of 0 and 14 years were enrolled in the study, 8 patients withdrew during the study and 21 patients completed the study.

Participant flow

Adverse events

Outcome measures

Plan to share IPD

IPD sharing Plan description

Recruitment status

No longer recruiting

Date of protocol fixation

2016

Year

07

Month

28

Day

Date of IRB

2016

Year

07

Month

28

Day

Anticipated trial start date

2016

Year

08

Month

10

Day

Last follow-up date

2023

Year

03

Month

31

Day

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Registered date

2016

Year

08

Month

01

Day

Last modified on

2024

Year

08

Month

07

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000027002

Single case data URL

Value
https://center6.umin.ac.jp/ice/27002