UMIN-CTR Clinical Trial

Basic information
Public title	Prospective, Multicenter, Observational Study to Evaluate Physical Activity, Bleeding Events, Quality of Daily Life, and Safety in Patients with Congenital Hemophilia A Without FVIII Inhibitors Under Emicizumab Prophylaxis
Acronym	TSUBASA Study An Observational Study of Emicizumab, a Bispecific Antibody recognizing coagulation factors IX and X, on Sports and physical Activities of people with haemophilia without FVIII inhibitors
Scientific Title	Prospective, Multicenter, Observational Study to Evaluate Physical Activity, Bleeding Events, Quality of Daily Life, and Safety in Patients with Congenital Hemophilia A Without FVIII Inhibitors Under Emicizumab Prophylaxis
Scientific Title:Acronym	TSUBASA Study An Observational Study of Emicizumab, a Bispecific Antibody recognizing coagulation factors IX and X, on Sports and physical Activities of people with haemophilia without FVIII inhibitors
Region	Japan

Condition
Condition	Congenital hemophilia A
Classification by specialty	Hematology and clinical oncology Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	1.To document, in an exploratory manner, the relationship between exercise status and bleeds in patients on prophylactic treatment with recombinant emicizumab. 2.To evaluate, in an exploratory manner, the quality and content of daily life during prophylactic treatment with emicizumab. 3.To collect information on the safety of emicizumab in clinical settings.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	1.Endpoints Related to Exercise Status and Bleeds:Patient-reported exercise status. Patient-reported status of bleeds attributable to exercise. Data of physical activity during exercise from wearable activity tracker. 2.Endpoints Related to Quality and Nature of Daily Life:Patient Health Related Quality of Life (HRQoL) determined with SF-36 scores or J-KIDSCREEN-52 scores. Work/study status determined with WPAI+CIQ:HS. Survey of physical activity with IPAQ. Questionnaire-based survey on daily life of patients and caregivers Data of non-exercise physical activity from wearable activity tracker. 3.Endpoints Related to Safety:Overall evaluation of the following measures on safety during emicizumab prophylactic treatment. -Adverse events, Presence of FVIII inhibitors
Key secondary outcomes

Base
Study type	Observational

Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
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) Informed consent form signed by the patient or legally acceptable representative Assent to participate in the study with signed informed assent form from pediatric patients when possible. (2) Diagnosis of congenital hemophilia A without FVIII inhibitors at the time of enrollment. (3) Patients for whom emicizumab treatment is selected as the most appropriate form of care (patients scheduled to receive emicizumab treatment in physician-patient treatment selection) and who are scheduled to undergo treatment per the latest package insert.
Key exclusion criteria	(1) Inherited or acquired bleeding disorder other than congenital hemophilia A. (2) Undergoing immune tolerance induction (ITI) at the time of enrollment. (3) Previous use of emicizumab. (4) Any other reason that, in the judgment of the investigator, would render the patient unsuitable for study participation.
Target sample size	160

Research contact person
Name of lead principal investigator	1st name Kagehiro / Keiji / Teruhisa Middle name Last name Amano / Nogami / Fujii
Organization	Tokyo Medical University / Nara Medical University / Hiroshima University Hospital
Division name	Department of Laboratory Medicine / Department of Pediatrics / Division of Blood Transfusion&Hemophilia Treatment Center
Zip code	1600023, 6348522, 7348551
Address	Nishishinjuku 6-7-1,Shinjuku-ku,Tokyo,Japan / Shijo-cho 840, Kashihara,Nara,Japan / Kasumi 1-2-3,Minami-ku,Hiroshima,Japan
TEL	(03-3342-6111)(0744-22-3051)(082-257-5555)
Email	cmastudy@chugai-pharm.co.jp

Public contact
Name of contact person	1st name the TSUBASA Study Middle name Last name The secretariat of
Organization	EP-CRSU Co.,Ltd.
Division name	Clinical Research Headquarters
Zip code	1620814
Address	Acropolis TOKYO,6-29Shinogawamachi,Shinjuku-ku,Tokyo,162-0814,JAPAN
TEL	03-5804-5045
Homepage URL
Email	prj-tsubasa@eps.co.jp

Sponsor
Institute	Chugai Pharmaceutical Co., Ltd.
Institute
Department

Funding Source
Organization	Chugai Pharmaceutical Co., Ltd.
Organization
Division
Category of Funding Organization	Profit organization
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization	Deliberates at each ethics committee of participating site
Address	Deliberates at each ethics committee of participating site
Tel	-
Email	cmastudy@chugai-pharm.co.jp

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	2019 Year 08 Month 01 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
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

Progress
Recruitment status	Open public recruiting
Date of protocol fixation	2019 Year 06 Month 20 Day
Date of IRB	2019 Year 06 Month 20 Day
Anticipated trial start date	2019 Year 11 Month 01 Day
Last follow-up date	2023 Year 10 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other

Other related information

(Email of lead principal investigator) Kagehiro Amano:kamano@tokyo-med.ac.jp Keiji Nogami:roc-noga@naramed-u.ac.jp Teruhisa Fujii:teruchan@hiroshima-u.ac.jp The study is a non-interventional, unblinded, non-randomized, single-arm, observational study to be conducted under clinical settings that will use healthcare information obtained as the result of providing care most appropriate to patients with no restrictions on the use and extent of tests, medications, and other medical acts performed for diagnostic or therapeutic reasons.

Management information
Registered date	2019 Year 07 Month 22 Day
Last modified on	2020 Year 07 Month 01 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000042682

Research Plan
Registered date	File name

Research case data specifications
Registered date	File name

Research case data
Registered date	File name

Contact us.