UMIN-ICDS Clinical Trial
| Unique ID issued by UMIN | UMIN000061017 |
|---|---|
| Receipt number | R000069824 |
| Scientific Title | Prospective observational study of HLA class I allele-deficient blood cells and PNH-type blood cells in newly diagnosed aplastic anemia |
| Date of disclosure of the study information | 2026/03/23 |
| Last modified on | 2026/03/23 12:16:08 |
* This page includes information on clinical trials registered in UMIN clinical trial registed system.
* We don't aim to advertise certain products or treatments
Basic information
Public title
Prospective study of immune escape clones in aplastic anemia
Acronym
ESCAPE-AA study
Scientific Title
Prospective observational study of HLA class I allele-deficient blood cells and PNH-type blood cells in newly diagnosed aplastic anemia
Scientific Title:Acronym
ESCAPE-AA
Region
| Japan |
Condition
Condition
Aplastic anemia
Classification by specialty
| Hematology and clinical oncology |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
To determine whether the presence and longitudinal dynamics of HLA class I allele-deficient blood cells and PNH-type blood cells are associated with hematologic recovery (complete response) and clinical outcomes in patients with aplastic anemia.
Basic objectives2
Others
Basic objectives -Others
In patients with aplastic anemia, to longitudinally evaluate the proportions of HLA class I allele-deficient blood cells and PNH-type blood cells and to prospectively examine their associations with treatment response, survival, clonal evolution to MDS/AML, and development of PNH. In addition, comprehensive genomic analyses will be performed in a subset of patients to elucidate the molecular basis of clonal dynamics.
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Longitudinal changes in the proportions of HLA class I allele-deficient blood cells and PNH-type blood cells
Key secondary outcomes
Complete response rate at 1 year, overall response rate at 1 year, time to complete response, time to overall response, overall survival, relapse, clonal evolution to MDS/AML, and PNH development
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
| 2 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
Participants must meet all of the following criteria:
1. Patients with aplastic anemia aged 2 years or older at the time of enrollment
2. Platelet count <100,000/uL
3. Have undergone bone marrow examination (smear, cytogenetic analysis, and biopsy) within 6 months prior to enrollment
4. Evidence of bone marrow hypoplasia, defined as bone marrow cellularity <30% on biopsy or reduced hematopoietic activity on MRI
Key exclusion criteria
Participants meeting any of the following criteria will be excluded:
1. Prior treatment with anti-thymocyte globulin (ATG)
2. Ongoing treatment for >=6 months prior to enrollment with immunosuppressive agents (e.g., cyclosporine) or thrombopoietin receptor agonists
3. History of chemotherapy or radiotherapy within the past 5 years
4. Presence of cytogenetic abnormalities specific to hematologic malignancies, including complex karyotype, -7, 7q-, or der(1;7), on bone marrow cytogenetic analysis
5. Increased blasts, defined as persistent >=1% in peripheral blood or >=5% in bone marrow
6. Normocellular to hypercellular megakaryopoiesis observed in either bone marrow smear or biopsy
7. No elevation of serum thrombopoietin levels (only in patients in whom this was measured)
8. History of cytopenia for >=2 years prior to enrollment (defined as neutrophil count <1,500/uL [or white blood cell count <3,000/uL], hemoglobin <11 g/dL, or platelet count <150,000/uL), unless attributable to a clearly benign cause such as iron deficiency anemia
9. Secondary bone marrow failure syndromes (e.g., bone marrow carcinomatosis, myelofibrosis, hemophagocytic syndrome, systemic lupus erythematosus, drug-induced agranulocytosis)
10. Inherited bone marrow failure syndromes (including suspected cases)
11. Concomitant hematologic malignancies (hypoplastic myelodysplastic syndrome is not excluded unless the above criteria are met)
12. Concomitant solid tumors or autoimmune diseases requiring systemic treatment
13. Uncontrolled infections refractory to antimicrobial therapy
14. Life expectancy of <1 year due to comorbidities other than aplastic anemia
15. Inability to attend regular follow-up visits at a hematology outpatient clinic
Target sample size
500
Research contact person
Name of lead principal investigator
| 1st name | Yoshitaka |
| Middle name | |
| Last name | Zaimoku |
Organization
Kanazawa University Hospital
Division name
Innovative Clinical Research Center
Zip code
920-8641
Address
13-1 Takaramachi, Kanazawa, Ishikawa
TEL
076-265-2090
zmk@staff.kanazawa-u.ac.jp
Public contact
Name of contact person
| 1st name | Yoshitaka |
| Middle name | |
| Last name | Zaimoku |
Organization
Kanazawa University Hospital
Division name
Innovative Clinical Research Center
Zip code
920-8641
Address
13-1 Takaramachi, Kanazawa, Ishikawa
TEL
076-265-2090
Homepage URL
zmk@staff.kanazawa-u.ac.jp
Sponsor or person
Institute
Kanazawa University
Institute
Department
Personal name
Funding Source
Organization
Ministry of Education, Culture, Sports, Science and Technology
Organization
Division
Category of Funding Organization
Japanese Governmental office
Nationality of Funding Organization
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Human Genome/Gene Analysis Research Ethics Committee of Kanazawa University
Address
13-1 Takaramachi, Kanazawa, Ishikawa
Tel
076-265-2110
rinri@adm.kanazawa-u.ac.j
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
金沢大学附属病院（石川県）、北見赤十字病院（北海道）、社会医療法人北楡会 札幌北楡病院（北海道）、北海道大学病院（北海道）、岩手医科大学附属病院（岩手県）、東北大学病院（宮城県）、平鹿総合病院（秋田県）、弘前大学医学部附属病院（青森県）、亀田総合病院（千葉県）、筑波大学附属病院（茨城県）、獨協医科大学病院（栃木県）、同愛記念病院（東京都）、東京科学大学（東京都）、順天堂大学医学部附属順天堂医院（東京都）、東京大学医学部附属病院（東京都）、がん・感染症センター都立駒込病院（東京都）、虎の門病院（東京都）、NTT東日本関東病院（東京都）、慶應義塾大学病院（東京都）、自治医科大学附属さいたま医療センター（埼玉県）、順天堂大学医学部附属練馬病院（東京都）、TMGあさか医療センター（埼玉県）、済生会横浜市南部病院（神奈川県）、東京慈恵会医科大学附属第三病院（東京都）、防衛医科大学校病院（埼玉県）、国家公務員共済組合連合会立川病院（東京都）、北里大学病院（神奈川県）、東京薬科大学ゲノム情報医科学研究室（東京都）、埼玉医科大学国際医療センター（埼玉県）、埼玉医科大学病院（埼玉県）、群馬大学医学部附属病院（群馬県）、聖隷浜松病院（静岡県）、浜松医療センター（静岡県）、富山県立中央病院（富山県）、富山市立富山市民病院（富山県）、富山赤十字病院（富山県）、愛知医科大学病院（愛知県）、厚生連高岡病院（富山県）、社会医療法人財団董仙会恵寿総合病院（石川県）、市立砺波総合病院（富山県）、名古屋大学大学院医学系研究科（愛知県）、江南厚生病院（愛知県）、金沢医療センター（石川県）、恵寿金沢病院（石川県）、JCHO金沢病院（石川県）、石川県立中央病院（石川県）、公立松任石川中央病院（石川県）、小松市民病院（石川県）、福井大学医学部附属病院（福井県）、福井県済生会病院（福井県）、福井赤十字病院（福井県）、岡波総合病院（愛知県）、京都大学医学部附属病院（京都府）、京都第一赤十字病院（京都府）、京都府立医科大学（京都府）、京都第二赤十字病院（京都府）、近畿大学奈良病院（奈良県）、大阪大学医学部附属病院（大阪府）、大阪国際がんセンター（大阪府）、大阪公立大学大学院医学研究科（大阪府）、神戸市立医療センター中央市民病院（兵庫県）、和歌山県立医科大学（和歌山県）、神戸市立西神戸医療センター（兵庫県）、岡山大学病院（岡山県）、愛媛大学医学部附属病院（愛媛県）、島根大学（島根県）、JCHO徳山中央病院（山口県）、宮崎大学医学部附属病院（宮崎県）、山口大学医学部附属病院（山口県）、地域医療機能推進機構九州病院（福岡県）、浜の町病院（福岡県）、佐賀大学医学部附属病院（佐賀県）、長崎大学病院（長崎県）、帝京大学医学部附属溝口病院（神奈川県）、国立国際医療研究センター病院（東京都）、愛育病院（東京都）、市立青梅総合医療センター（東京都）、横浜市立大学附属病院（神奈川県）、香川大学医学部附属病院（香川県）、三重大学医学部附属病院（三重県）、国際医療福祉大学三田病院（東京都）、長野赤十字病院（長野県）、大分大学医学部附属病院（大分県）、沖縄県立中部病院（沖縄県）
Other administrative information
Date of disclosure of the study information
| 2026 | Year | 03 | Month | 23 | 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
Open public recruiting
Date of protocol fixation
| 2024 | Year | 09 | Month | 30 | Day |
Date of IRB
| 2024 | Year | 10 | Month | 15 | Day |
Anticipated trial start date
| 2024 | Year | 10 | Month | 15 | Day |
Last follow-up date
| 2029 | Year | 03 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
This is a multicenter prospective observational study. The proportions of HLA class I allele-deficient blood cells and PNH-type blood cells will be measured by flow cytometry at enrollment and at predefined time points (1, 3, and 6 months after enrollment, and annually thereafter). Clinical data, including laboratory findings, treatment, response, survival, clonal evolution, and development of PNH, will also be collected. Comprehensive genomic analyses will be performed in a subset of patients..
Management information
Registered date
| 2026 | Year | 03 | Month | 23 | Day |
Last modified on
| 2026 | Year | 03 | Month | 23 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000069824
