UMIN-ICDS Clinical Trial
| Unique ID issued by UMIN | UMIN000062334 |
|---|---|
| Receipt number | R000071123 |
| Scientific Title | A Retrospective Observational Study Evaluating Treatment Response in Patients with PNH on Anti-C5 Antibody Therapy: Japanese Chart Review Study |
| Date of disclosure of the study information | 2026/07/31 |
| Last modified on | 2026/07/24 16:28:16 |
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Basic information
Public title
A Retrospective Observational Study Evaluating Treatment Response in Patients with PNH on Anti-C5 Antibody Therapy: Japanese Chart Review Study
Acronym
ROSETTA-J
Scientific Title
A Retrospective Observational Study Evaluating Treatment Response in Patients with PNH on Anti-C5 Antibody Therapy: Japanese Chart Review Study
Scientific Title:Acronym
ROSETTA-J
Region
| Japan |
Condition
Condition
Paroxysmal Nocturnal Hemoglobinuria
Classification by specialty
| Hematology and clinical oncology |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
Although anti-C5 antibody therapy has improved treatment outcomes in PNH, anemia and hemolysis-related symptoms may persist in some patients with PNH. This study aimed to characterize real-world treatment responses to anti-C5 antibody therapy in Japan and to identify factors associated with suboptimal responses, thereby clarifying remaining unmet needs and opportunities for treatment optimization.
Basic objectives2
Others
Basic objectives -Others
To characterize patients with an inadequate treatment response to anti-C5 antibody therapy and to clarify unmet needs in the clinical management of PNH in Japan.
Trial characteristics_1
Exploratory
Trial characteristics_2
Others
Developmental phase
Not applicable
Assessment
Primary outcomes
The proportion of patients with PNH in Japan who initiated anti-C5 antibody therapy and had a hemoglobin level of less than 12 g/dL at 6 months after initiation of anti-C5 antibody therapy
Key secondary outcomes
1) Distribution of patients with Hb levels less than 12 g/dL at 6 months after initiation of anti-C5 antibody therapy
2) Changes in clinical laboratory values, including Hb, after initiation of anti-C5 antibody therapy.
3) Number of red blood cell transfusions after initiation of anti-C5 antibody therapy.
4) Incidence of cholelithiasis after initiation of anti-C5 antibody therapy, time to first occurrence, and estimated incidence rate from treatment initiation to the date of the final clinic visit).
5) Incidence of EVH after initiation of anti-C5 antibody therapy, time to first occurrence, and estimated incidence rate.
6) Number of occurrences of hemolytic events , including BTH, exacerbation of EVH, after initiation of anti-C5 antibody therapy, time to first occurrence, and estimated incidence rate.
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
| 18 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
1) Patients who were diagnosed with PNH at the participating institution and were 18 years of age or older at the initiation of anti-C5 antibody therapy, defined as the index date
2) Patients who had a confirmed diagnosis of PNH before the index date based on flow cytometry and LDH levels
3) PNH patients who had continued anti-C5 antibody therapy for at least 6 months, or who had previously received anti-C5 antibody therapy for PNH for at least 6 months
4) Patients who had at least two Hb measurements during the observation period after the index date, with at least one Hb measurement performed within +- 1 month of 6 months after the index date
Key exclusion criteria
1) Patients known or suspected to have a C5 gene polymorphisms when treated with an anti-C5 antibody that recognizes the C5 MG7 domain
2) Patients who participated in any clinical trial within 6 months after the index date
3) Any other patients deemed ineligible by the principal investigator or sub-investigator
Target sample size
70
Research contact person
Name of lead principal investigator
| 1st name | Jun-ichi |
| Middle name | |
| Last name | Nishimura |
Organization
Japan PNH Study Group
Division name
PNH Research Society Secretariat
Zip code
103-0025
Address
2-17-5 Nihombashi Kayabacho, Chuo-ku, Tokyo
TEL
03-5651-7105
study@pnh.or.jp
Public contact
Name of contact person
| 1st name | Osamu |
| Middle name | |
| Last name | Ishihara |
Organization
Japan PNH Study Group
Division name
Office for Clinical Research
Zip code
103-0025
Address
2-17-5 Nihombashi Kayabacho, Chuo-ku, Tokyo
TEL
03-5651-7105
Homepage URL
study@pnh.or.jp
Sponsor or person
Institute
Japan PNH Study Group / Novartis Pharma K.K.
Institute
Department
Personal name
Funding Source
Organization
Novartis Pharma K.K.
Organization
Division
Category of Funding Organization
Profit organization
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Nihonbashi Sakura Clinic Ethics Review Committee
Address
1-9-2 Nihombashi Kayabacho, Chuo-ku, Tokyo
Tel
03-6661-9061
c-irb_ug@neues.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
国立大学法人大阪大学（大阪府）/ Osaka University
国立大学法人熊本大学（熊本県）/ Kumamoto University
国立大学法人筑波大学（茨城県）/ University of Tsukuba
学校法人慶應義塾大学（東京都）/ Keio University
国立大学法人金沢大学（石川県）/ Kanazawa University
国立大学法人東北大学（宮城県）/ Tohoku University
学校法人東京医科大学（東京都）/ Tokyo Medical University
学校法人自治医科大学（栃木県）/ Jichi Medical University
公立大学法人福島県立医科大学（福島県）/ Fukushima Prefectural Medical University
国立大学法人京都大学（京都府）/ Kyoto University
一般財団法人住友病院（大阪府）/ Sumitomo Hospital
公益社団法人地域医療振興協会 練馬光が丘病院（東京都）/ Nerima Hikarigaoka Hospital
Other administrative information
Date of disclosure of the study information
| 2026 | Year | 07 | Month | 31 | 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
67
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
No longer recruiting
Date of protocol fixation
| 2025 | Year | 05 | Month | 13 | Day |
Date of IRB
| 2025 | Year | 05 | Month | 21 | Day |
Anticipated trial start date
| 2025 | Year | 10 | Month | 08 | Day |
Last follow-up date
| 2025 | Year | 12 | Month | 31 | Day |
Date of closure to data entry
| 2026 | Year | 01 | Month | 15 | Day |
Date trial data considered complete
| 2026 | Year | 05 | Month | 15 | Day |
Date analysis concluded
Other
Other related information
Analysis in progress
Management information
Registered date
| 2026 | Year | 07 | Month | 24 | Day |
Last modified on
| 2026 | Year | 07 | Month | 24 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000071123
