UMIN-ICDS Clinical Trial
| Unique ID issued by UMIN | UMIN000050211 |
|---|---|
| Receipt number | R000056855 |
| Scientific Title | THE CLINICAL, HUMANISTIC, AND ECONOMIC BURDEN AMONG PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) IN JAPAN |
| Date of disclosure of the study information | 2023/02/02 |
| Last modified on | 2023/10/21 16:06:36 |
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Basic information
Public title
THE CLINICAL, HUMANISTIC, AND ECONOMIC BURDEN AMONG PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) IN JAPAN
Acronym
THE CLINICAL, HUMANISTIC, AND ECONOMIC BURDEN AMONG PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) IN JAPAN
Scientific Title
THE CLINICAL, HUMANISTIC, AND ECONOMIC BURDEN AMONG PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) IN JAPAN
Scientific Title:Acronym
THE CLINICAL, HUMANISTIC, AND ECONOMIC BURDEN AMONG PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) IN JAPAN
Region
| Japan |
Condition
Condition
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Classification by specialty
| Hematology and clinical oncology |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
-To describe the clinical, humanistic, and economic burden of PNH on patients currently being treated with C5 inhibitors (either eculizumab or ravulizumab) in Japan
-To analyze the clinical, humanistic, and economic burden of PNH in patients currently being treated with C5 inhibitors by subgroup
Basic objectives2
Others
Basic objectives -Others
Web-based questionnaire survey and paper-based self-completion questionnaire survey on disease burden
Trial characteristics_1
Others
Trial characteristics_2
Others
Developmental phase
Not applicable
Assessment
Primary outcomes
Self-reported hematological and clinical measures (such as diagnosis of ongoing anemia, levels of hemoglobin, history of red blood cell transfusions, history of thrombotic events, and history of renal function impairment)
Quality of life assessed using PRO scales (EQ-5D-5L and EORTC)
Fatigue and depression state assessed using PRO scales (FACIT-Fatigue and PHQ-8)
Impact on work productivity assessed using a PRO scale (WPAI) and impact of C5 inhibitor infusions and blood transfusions assessed using a Likert scale
Self-reported health resource utilization (Number of times in ER and hospitalized)
Key secondary outcomes
Calculation of summary statistics and number and percentage of respondents for the primary endpoints by subgroup described below
-Subgroup
Patients with reported hemoglobin levels of <12 (120) vs >_12 (120) g/dL (g/L) and <10.5 (105) vs >_10.5 (105) g/dL (g/L)
Patients with vs. without a history of transfusion in the past 6 months
Patients with vs. without a history of thrombotic events
Patients classification as separately specified in the Statistical Analysis Plan (SAP)
Base
Study type
Others,meta-analysis etc
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
| 18 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
-18 years old or older
-Self-reported diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH)
-Self-reported that currently being treated with eculizumab or ravulizumab
-Agree to informed consent for the study
Key exclusion criteria
-Correspond to an inappropriate answer
Target sample size
60
Research contact person
Name of lead principal investigator
| 1st name | Tomoyuki |
| Middle name | - |
| Last name | Kusaka |
Organization
Social Survey Research Information co., Ltd(SSRI)
Division name
Healthcare BI/MA Service Division
Zip code
162-0067
Address
10-5 Tomihisacho, Shinjuku-ku, Tokyo 162-0067 JAPAN
TEL
03-6709-9710
hor@ssri.com
Public contact
Name of contact person
| 1st name | Moemi |
| Middle name | |
| Last name | Miura |
Organization
Social Survey Research Information co., Ltd(SSRI)
Division name
Health outcomes research, HBI/MA Service Division
Zip code
162-0067
Address
10-5 Tomihisacho, Shinjuku-ku, Tokyo 162-0067 JAPAN
TEL
03-6709-9710
Homepage URL
hor@ssri.com
Sponsor or person
Institute
Swedish Orphan Biovitrum Japan
Institute
Department
Personal name
Funding Source
Organization
Swedish Orphan Biovitrum Japan
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
Kitamachi clinic
Address
1-1-3 Kichijoji-Kitamachi, Musashino-city, Tokyo
Tel
03-6779-8166
chi-pr-ec-kitamachi@cmicgroup.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
| 2023 | Year | 02 | Month | 02 | 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
68
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
| 2022 | Year | 09 | Month | 13 | Day |
Date of IRB
| 2022 | Year | 09 | Month | 21 | Day |
Anticipated trial start date
| 2022 | Year | 09 | Month | 30 | Day |
Last follow-up date
| 2022 | Year | 11 | Month | 30 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
N/A
Management information
Registered date
| 2023 | Year | 02 | Month | 01 | Day |
Last modified on
| 2023 | Year | 10 | Month | 21 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000056855
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