UMIN-ICDS Clinical Trial

Public title

A retrospective chart review study of recombinant factor IX albutrepenonacog alfa treatment in previously untreated pediatric patients with hemophilia B in Japan

Acronym

A retrospective chart review study of recombinant factor IX albutrepenonacog alfa treatment in previously untreated pediatric patients with hemophilia B in Japan

Scientific Title

A retrospective chart review study of recombinant factor IX albutrepenonacog alfa treatment in previously untreated pediatric patients with hemophilia B in Japan

Scientific Title:Acronym

A retrospective chart review study of recombinant factor IX albutrepenonacog alfa treatment in previously untreated pediatric patients with hemophilia B in Japan

Region

Japan

Condition

Hemophilia B

Classification by specialty

Hematology and clinical oncology

Child

Classification by malignancy

Others

Genomic information

NO

Narrative objectives1

1) What are the real-world treatment patterns of recombinant factor IX (FIX)-albumin fusion protein (rIX-FP, IDELVION) in pediatric previously untreated patients (PUPs) with hemophilia B?
2) What are the patient background and baseline clinical characteristics of pediatric PUPs with hemophilia B?
3) What is the real-world effectiveness on bleeding events and safety on the incidence of FIX inhibitor development after initiation of treatment with rIX-FP?

Basic objectives2

Safety,Efficacy

Basic objectives -Others

Trial characteristics_1

Exploratory

Trial characteristics_2

Pragmatic

Developmental phase

Not applicable

Primary outcomes

1) To describe the real-world treatment patterns of rIX-FP in pediatric PUPs with hemophilia B
a. Treatment duration (days)
b. Total number of doses [i.e., Expousre days (EDs) to rIX-FP]
c. Total dosage (IU)
d. Mean number of doses per month
e. Dose per body weight (IU/kg)
f. Dosage interval (days)
g. Treatment regimen (prophylaxis, on-demand, perioperative)
h. Use of CV port (days)
i. Plasma FIX activity (%) during the treatment period
2) To describe the patient background and baseline clinical characteristics of pediatric PUPs with hemophilia B who initiated treatment with rIX-FP.
a. Sex (male, female)
b. Body weight (kg)
c. Age at diagnosis (months, years)
d. Age at initiation of rIX-FP (<2, 2<=to<6, 6<=to<12 years)
e. Severity (severe, plasma FIX activity<1%; moderate, 1%<=to<=5%; mild, 5%< to <40%; unknown)
f. Family history of hemophilia
g. Family history of inhibitor
h. IVR ([IU/dL]/[IU/kg])
i. History of bleeding events
j. Reason for visiting the facility (bleeding event, family history, others)
k. Comorbidities and medical history at baseline (before and at initiation of rIX-FP)

Key secondary outcomes

To describe the real-world effectiveness on bleeding (all bleeding, treatment required) events and safety on the incidence of FIX inhibitor development after initiation of treatment with rIX-FP.
a. Number of patients with bleeding events at follow-up
b. Number of bleeding events at follow-up
- all bleeding, treatment required, bleeding type (spontaneous, traumatic, surgical, others)
c. Number of bleeding events per patient and annualized bleeding rate (ABR) at follow-up
d. Number of patients with FIX inhibitor development at follow-up
e. Allergic drug reactions

Study type

Observational

Basic design

Randomization

Randomization unit

Blinding

Control

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

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

Age-lower limit

Not applicable

Age-upper limit

12

years-old

>

Gender

Male and Female

Key inclusion criteria

1) Pediatric patients with a diagnosis of hemophilia B who were <12 years of age at initiation of rIX-FP.
2) Patients who initiated rIX-FP between 1 May 2017 and 30 September 2023 as prophylaxis, on-demand, and/or perioperative treatment(s) according to the label and continued to use rIX-FP for >6 months or had any activity record in medical chart for >6 months.
3) Provision of the patient's or the legally acceptable representative's informed consent or assent in pediatric research according to the pediatric patient's capacity to consent.
In cases of difficulty in obtaining written consent due to transfer to a different hospital, death, or other reasons, only if the central ethics review committee permits opt-out, in which the patients or the legally acceptable representatives can access the opt-out information and are given the opportunity to refuse the provision of his/her existing information, enrollment of the patient will be permitted.

Key exclusion criteria

1) Participating in any interventional clinical trials during the period between 1 May 2017 and 30 June 2024.
If patients discontinue rIX-FP during the period above, patients who participated in any interventional clinical trials during the period between 1 May 2017 and the date of discontinuation will be excluded from the study.

* Treatment discontinuation is defined as: (1) a switch to other FIX products (excluding a temporary switch defined as <3 doses of other FIX products/<1 month duration of other FIX products) and (2) loss to follow up due to transfer to a different hospital, death, or other reasons. The date of discontinuation is defined as (1) the date of a switch to other FIX products, (2)the date of transfer to a different hospital, the date of death, or the date of loss to follow-up confirmed by the investigator.

2) Patients who have previously treated with other FIX product(s) before initiation of rIX-FP
3) Patients enrolled in rIX-FP post-marketing surveillance (PMS)

Target sample size

24

Name of lead principal investigator

1st name	Keiji
Middle name
Last name	Nogami

Organization

Nara Medical University

Division name

Department of Pediatrics,

Zip code

634-8521

Address

840 Shijo-Cho, Kashihara, Nara, Japan

TEL

0744-22-3051

Email

roc-noga@naramed-u.ac.jp

Name of contact person

1st name	Yuki
Middle name
Last name	Murabayashi

Organization

Mebix, Inc.

Division name

Research Promotion Headquarters

Zip code

107-0052

Address

Akasaka Intercity, 1-11-44 Akasaka, Minato-ku, Tokyo

TEL

03-4362-4500

Homepage URL

Email

idelvion_chart_review@mebix.co.jp

Institute

CSL Behring K.K.

Institute

Department

Personal name

Naoki Terasaka

Organization

CSL Behring K.K.

Organization

Division

Category of Funding Organization

Profit organization

Nationality of Funding Organization

Co-sponsor

Name of secondary funder(s)

Organization

Non-Profit Organization MINS Research Ethics Committee

Address

5-20-9-401 Mita, Minato-ku, Tokyo

Tel

03-6416-1868

Email

npo-mins@j-irb.com

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

Date of disclosure of the study information

2024

Year

02

Month

28

Day

URL releasing protocol

Publication of results

Unpublished

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

Recruitment status

Preinitiation

Date of protocol fixation

2024

Year

01

Month

10

Day

Date of IRB

2024

Year

02

Month

08

Day

Anticipated trial start date

2024

Year

05

Month

01

Day

Last follow-up date

2024

Year

10

Month

31

Day

Date of closure to data entry

2024

Year

10

Month

31

Day

Date trial data considered complete

Date analysis concluded

Other related information

This study is a multicenter, retrospective, medical chart review to reveal the real-world data of pediatric PUPs with hemophilia B who initiated rIX-FP in Japan, including treatment patterns, patient background, effectiveness on bleeding events, and safety on inhibitor development. This study will be conducted in pediatric PUPs with a diagnosis of hemophilia B who were <12 years of age at initiation of rIX-FP, initiated rIX-FP during the period between 1 May 2017 and 30 September 2023 as prophylaxis, on-demand, and/or perioperative treatment(s) according to the label, and continued >6 months of the rIX-FP treatment or had >6 months of any activity records in the medical chart. The patients' medical chart data before 30 June 2024 will be collected retrospectively.
The medical chart data of interest in this study include injection records of rIX-FP [e.g., treatment regimen (prophylaxis, on-demand, perioperative), treatment data on prophylaxis (treatment duration, dosage interval, dose), treatment data on on-demand and perioperative, data on plasma FIX activity, use of CV port], patient background [e.g., sex, month and year of birth, severity of disease (plasma FIX activity), family history of hemophilia and inhibitor, IVR, history of bleeding events, reasons for visiting the facility (bleeding events, family history, others), comorbidities and medical history at baseline (before and at initiation of rIX-FP)], number of bleeding for all and treatment required bleeding events and for bleeding type (spontaneous, traumatic, surgical, others), and the safety data on inhibitor development and allergic drug reactions to rIX-FP.

Registered date

2024

Year

02

Month

27

Day

Last modified on

2024

Year

02

Month

27

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000061129