UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Sirolimus for the treatment of lymphangiomatosis, Gorham-Stout disease and vascular anomalies
Title of the study (Brief title)	Sirolimus for vascular anomalies
Region	Japan

Condition
Condition	Lymphangiomatosis, Gorham-Stout disease and vascular anomalies
Classification by specialty	Medicine in general Gastroenterology Hepato-biliary-pancreatic medicine Cardiology Pneumology Hematology and clinical oncology Surgery in general Chest surgery Pediatrics Orthopedics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the efficacy of sirolimus for lymphangiomatosis, Gorham-Stout disease and vascular anomalies
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2
Developmental phase	Phase I,II

Assessment
Primary outcomes	Improvement of clinical symptoms at 1, 3, 6, 12 after administration
Key secondary outcomes

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	Oral sirolimus
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	0 years-old <=
Age-upper limit	30 years-old >=
Gender	Male and Female
Key inclusion criteria	The patients with intaracbtable lymphangiomatosis, Gorham-Stout disease and vascular anomalies
Key exclusion criteria	Allergy of sirolimus
Target sample size	30

Research contact person
Name of lead principal investigator	Michio Ozeki
Organization	Gifu University
Division name	Pediatrics
Address	1-1, Yanagito, Gifu-shi, Gifu
TEL	058-230-6000
Email	michioo@gifu-u.ac.jp

Public contact
Name of contact person	Michio Ozeki
Organization	Gifu University
Division name	Pediatrics
Address	1-1, Yanagito, Gifu-shi, Gifu
TEL	058-230-6000
Homepage URL
Email	michioo@gifu-u.ac.jp

Sponsor
Institute	Gifu university
Institute
Department

Funding Source
Organization	Gifu university
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

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

Other administrative information
Date of disclosure of the study information	2015 Year 02 Month 19 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2015 Year 02 Month 01 Day
Anticipated trial start date	2015 Year 02 Month 01 Day
Last follow-up date	2020 Year 02 Month 01 Day
Date of closure to data entry	2020 Year 02 Month 01 Day
Date trial data considered complete	2020 Year 02 Month 01 Day
Date analysis concluded	2022 Year 02 Month 01 Day

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2015 Year 02 Month 19 Day
Last modified on	2018 Year 01 Month 02 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000019237