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Recruitment status Open public recruiting
Unique ID issued by UMIN UMIN000037448
Receipt No. R000042682
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
Date of disclosure of the study information 2019/08/01
Last modified on 2020/07/01

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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


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