UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Efficacy and safety of low dose sirolimus in lymphangioleiomyomatosis
Title of the study (Brief title)	Low dose sirolimus study
Region	Japan

Condition
Condition	lymphangioleiomyomatosis
Classification by specialty	Pneumology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	Safety
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	1) The FEV1 response, which is assessed as the rate of change in FEV1 or the SGRQ score response. 2) Adverse events in sirolimus 1mg/day and 2mg/day.
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	Dose comparison
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	sirolimus 1mg/day / sirolimus 2mg/day
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	Not applicable
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1) Diagnosis of LAM 2) The first treatment of sirolimus or no treatment of sirolimus for 12 weeks
Key exclusion criteria	1) Pregnancy or planning of pregnancy or lactating 2) Severe dysemia or impairment of liver function 3) Active infection 4) Operation within 8 weeks 5) Severe dyslipidemia 6) Active registration of lung transplantation 7) Allergy of sirolimus
Target sample size	20

Research contact person
Name of lead principal investigator	Yoshikazu Inoue
Organization	National Hospital Organization Kinki-Chuo Chest Medical Center
Division name	Clinical Research Center
Address	1180, Nagasone-cho Kita-ku, Sakai-city, Osaka, 591-8555, Japan
TEL	072-252-3021
Email	giichi@kch.hosp.go.jp

Public contact
Name of contact person	Chikatoshi Sugimoto
Organization	National Hospital Organization Kinki-Chuo Chest Medical Center
Division name	Clinical Research Center
Address	1180, Nagasone-cho Kita-ku, Sakai-city, Osaka, 591-8555, Japan
TEL	072-252-3021
Homepage URL
Email	sugimoto@kch.hosp.go.jp

Sponsor
Institute	National Hospital Organization Kinki-chuo Chest Medical Center
Institute
Department

Funding Source
Organization	National Hospital Organization Kinki-chuo Chest Medical Center
Organization
Division
Category of Funding Organization	Other
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 03 Month 02 Day

Progress
Recruitment status	Enrolling by invitation
Date of protocol fixation	2014 Year 12 Month 25 Day
Anticipated trial start date	2015 Year 01 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

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

Management information
Registered date	2015 Year 03 Month 02 Day
Last modified on	2019 Year 03 Month 04 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000019250