UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000044800 |
|---|---|
| Receipt number | R000051164 |
| Scientific Title | An Observational Study to Investigate the Effectiveness of PK Guided Personalized Recombinant (r)FVIII Treatment in Patients with Hemophilia A |
| Date of disclosure of the study information | 2021/07/09 |
| Last modified on | 2024/07/05 09:40:37 |
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Basic information
Public title
An Observational Study to Investigate the Effectiveness of PK Guided Personalized Recombinant (r)FVIII Treatment in Patients with Hemophilia A
Acronym
An Observational Study to Investigate the Effectiveness of PK Guided Personalized Recombinant (r)FVIII Treatment in Patients with Hemophilia A
Scientific Title
An Observational Study to Investigate the Effectiveness of PK Guided Personalized Recombinant (r)FVIII Treatment in Patients with Hemophilia A
Scientific Title:Acronym
An Observational Study to Investigate the Effectiveness of PK Guided Personalized Recombinant (r)FVIII Treatment in Patients with Hemophilia A
Region
| Japan |
Condition
Condition
Hemophilia A
Classification by specialty
| Hematology and clinical oncology | Pediatrics |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To explore and evaluate the utility of myPKFiT in male patients with hemophilia A receiving personalized treatment with myPKFiT with rurioctocog alfa or rurioctocog alfa pegol
Basic objectives2
Others
Basic objectives -Others
This is a multicenter, exploratory, observational study in male patients with hemophilia A undergoing personalized treatment with myPKFiT using either rurioctocog alfa or rurioctocog alfa pegol to evaluate ABR, ABR by each of the following details of bleeding (traumatic hemorrhage, spontaneous hemorrhage, joint hemorrhage, non-joint hemorrhage), rFVIII product consumption, physical activity level, QOL, myPKFiT mobile application satisfaction, target and observed trough levels, and number of rFVIII administrations per week in 6 months before and after adjustment using myPKFiT.
Trial characteristics_1
Others
Trial characteristics_2
Others
Developmental phase
Not applicable
Assessment
Primary outcomes
Primary endpoint
Annualized bleeding rate (ABR)
Key secondary outcomes
<Secondary endpoints>
1.ABR by each of the following details of bleeding (traumatic hemorrhage, spontaneous hemorrhage, joint hemorrhage, non-joint hemorrhage)
2.Consumption of rFVIII products
3.Amount of physical activity
4.QOL
5.Satisfaction with the myPKFiT mobile application (only for users)
<Exploratory evaluations>
1.Target and observed trough (only if evaluable)
2.Number of rFVIII administrations per week
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
Key inclusion criteria
1. Male patients with congenital severe/moderate hemophilia A (severe= FVIII:C <1% at baseline, moderate*= FVIII: 1% <= C < 5% at baseline)
* : Patients receiving regular replacement therapy
2. Patients receiving or who had been treated with rurioctocog alfa and rurioctocog alfa pegol
3. Patients undergoing PK-guided personalized therapy (rFVIII replacement therapy) with myPKFiT
4. Age >=12 years at the time of initiation of myPKFiT (if on rurioctocog alfa pegol treatment)
Key exclusion criteria
1. Patients using PK guided tools that are unapproved in Japan
2. Patients on FVIII inhibitors
Target sample size
30
Research contact person
Name of lead principal investigator
| 1st name | Yasuo |
| Middle name | |
| Last name | Miyaguchi |
Organization
Takeda Pharmaceutical Company Limited.
Division name
Japan Medical Office
Zip code
103-8668
Address
1-1, Nihonbashi-Honcho 2-Chome, Chuo-ku, Tokyo, 103-8668, Japan
TEL
070-4921-7966
yasuo.miyaguchi@takeda.com
Public contact
Name of contact person
| 1st name | Yasuo |
| Middle name | |
| Last name | Miyaguchi |
Organization
Takeda Pharmaceutical Company Limited.
Division name
Japan Medical Office
Zip code
103-8668
Address
1-1, Nihonbashi-Honcho 2-Chome, Chuo-ku, Tokyo, 103-8668, Japan
TEL
070-4921-7966
Homepage URL
yasuo.miyaguchi@takeda.com
Sponsor or person
Institute
Takeda Pharmaceutical Company Limited.
Institute
Department
Personal name
Funding Source
Organization
Takeda Pharmaceutical Company Limited.
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
Specified Nonprofit Corporation MINS
Address
5-20-9-401 Mita, Minato-ku, Tokyo
Tel
03-6416-1868
npo-mins@j-irb.com
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
| 2021 | Year | 07 | Month | 09 | 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
7
Results
Results date posted
Results Delayed
| Delay expected |
Results Delay Reason
It takes time to publish the result into a paper.
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
| 2021 | Year | 06 | Month | 22 | Day |
Date of IRB
| 2021 | Year | 07 | Month | 08 | Day |
Anticipated trial start date
| 2021 | Year | 08 | Month | 20 | Day |
Last follow-up date
| 2022 | Year | 02 | Month | 20 | Day |
Date of closure to data entry
| 2022 | Year | 11 | Month | 30 | Day |
Date trial data considered complete
| 2022 | Year | 11 | Month | 30 | Day |
Date analysis concluded
| 2023 | Year | 03 | Month | 31 | Day |
Other
Other related information
NA
Management information
Registered date
| 2021 | Year | 07 | Month | 08 | Day |
Last modified on
| 2024 | Year | 07 | Month | 05 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000051164
