UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000028834
Receipt number R000033001
Scientific Title Multicenter observation study to examine the usefulness of transgenic blood coagulation factor VIII Fc fusion protein (rFVIIIFc) preparation for domestic pediatric hemophilia A patients
Date of disclosure of the study information 2017/09/01
Last modified on 2023/03/07 14:53:04

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Basic information

Public title

Multicenter observation study to examine the usefulness of transgenic blood coagulation factor VIII Fc fusion protein (rFVIIIFc) preparation for domestic pediatric hemophilia A patients

Acronym

Fc Adolescent and Children Treatment study (FACTs)

Scientific Title

Multicenter observation study to examine the usefulness of transgenic blood coagulation factor VIII Fc fusion protein (rFVIIIFc) preparation for domestic pediatric hemophilia A patients

Scientific Title:Acronym

Fc Adolescent and Children Treatment study (FACTs)

Region

Japan


Condition

Condition

hemophilia A

Classification by specialty

Hematology and clinical oncology

Classification by malignancy

Others

Genomic information

NO


Objectives

Narrative objectives1

Study the usefulness of rFVIIIFc for children with haemophilia (hemophilia A) based on real clinical practice in Japan

Basic objectives2

Efficacy

Basic objectives -Others


Trial characteristics_1


Trial characteristics_2


Developmental phase



Assessment

Primary outcomes

Number of annual spontaneous bleeding episodes over 2 years from registration

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

Blood coagulation preparation rFVIIIFc
over 2 years
25-65 IU/kg/dose
The dosing interval ranges from 3 to 5 days

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

18 years-old >

Gender

Male and Female

Key inclusion criteria

Hemophilia A patients who are planning to administer rFVIIIFc or have started administration within the past 12 months

Key exclusion criteria

Patients who are unable or unwilling to provide informed consent

Target sample size

3


Research contact person

Name of lead principal investigator

1st name Yasutomo
Middle name
Last name Funakoshi

Organization

Nagasaki University Hospital

Division name

Department of pediatrics

Zip code

853-8501

Address

1-7-1Sakamoto, Nagasaki

TEL

095-819-7298

Email

funakoshi@nagasaki-u.ac.jp


Public contact

Name of contact person

1st name Yasutomo
Middle name
Last name Funakoshi

Organization

Nagasaki University Hospital

Division name

Department of pediatrics

Zip code

852-8501

Address

1-7-1Sakamoto, Nagasaki

TEL

095-819-7298

Homepage URL


Email

funakoshi@nagasaki-u.ac.jp


Sponsor or person

Institute

Nagasaki University Hospital

Institute

Department

Personal name



Funding Source

Organization

Bioverativ Japan 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

Nagasaki University Hospital Clinical Research Ethics Committee

Address

1-7-1Sakamoto, Nagasaki

Tel

0958197229

Email

gaibushikin@ml.nagasaki-u.ac.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

2017 Year 09 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

Completed

Date of protocol fixation

2017 Year 08 Month 28 Day

Date of IRB

2017 Year 08 Month 22 Day

Anticipated trial start date

2017 Year 09 Month 01 Day

Last follow-up date

2022 Year 12 Month 31 Day

Date of closure to data entry


Date trial data considered complete


Date analysis concluded



Other

Other related information



Management information

Registered date

2017 Year 08 Month 25 Day

Last modified on

2023 Year 03 Month 07 Day



Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000033001


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name