UMIN-CTR Clinical Trial

Recruitment status Completed
Unique ID issued by UMIN UMIN000027482
Receipt No. R000031482
Scientific Title Complement activation and ADAMTS13 suppression may contribute to the characteristic pathological features in Upshaw-Schulman Syndrome
Date of disclosure of the study information 2017/06/01
Last modified on 2020/11/25 (Ver. 5)

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Basic information
Public title Complement activation and ADAMTS13 suppression may contribute to the characteristic pathological features in Upshaw-Schulman Syndrome
Acronym Complement activation and ADAMTS13 suppression in USS
Scientific Title Complement activation and ADAMTS13 suppression may contribute to the characteristic pathological features in Upshaw-Schulman Syndrome
Scientific Title:Acronym Complement activation and ADAMTS13 suppression in USS
Region
Japan

Condition
Condition Upshaw-Schulman syndrome
Classification by specialty
Hematology and clinical oncology Nephrology
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 To investigate the glomerular localization of ADAMTS13 and the commitment of complement activation of Upshaw-Schulman syndrome renal biopsies
Basic objectives2 Bio-availability
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes Immunohistochemistry of ADAMTS13, C4d and C5b-9 of Upshaw-Schulman syndrome renal biopsies
Key secondary outcomes

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 and Female
Key inclusion criteria Severe deficient ADAMTS13 activity
(<10%), negative for ADAMTS13 inhibitor and mutations in the ADAMTS13 gene
Key exclusion criteria Transplanted kidney
Target sample size 10

Research contact person
Name of lead principal investigator
1st name Hiroe
Middle name
Last name Itami
Organization Nara medical university
Division name Department of diagnostic pathology
Zip code 634-8521
Address 840 Shijo-Cho, Kashihara, Nara, Japan
TEL 0744-29-8910
Email hritami@naramed-u.ac.jp.

Public contact
Name of contact person
1st name Hiroe
Middle name
Last name Itami
Organization Nara medical university
Division name Department of diagnostic pathology
Zip code 634-8521
Address 840 Shijo-Cho, Kashihara, Nara, Japan
TEL 0744-29-8910
Homepage URL
Email hritami@naramed-u.ac.jp.

Sponsor
Institute Nara medical university
Institute
Department

Funding Source
Organization Nothing
Organization
Division
Category of Funding Organization Other
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization Nara medical university
Address 840 Shijo-Cho, Kashihara, Nara, Japan
Tel 0744-29-8910
Email hritami@naramed-u.ac.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

Other administrative information
Date of disclosure of the study information
2017 Year 06 Month 01 Day

Related information
URL releasing protocol None
Publication of results Published

Result
URL related to results and publications https://doi.org/10.1016/j.thromres.2018.08.020
Number of participants that the trial has enrolled 5
Results 
Chronic glomerular sclerotic changes in the majority of USS cases (4 of 5), with minor glomerular pathology in the remaining case. In two of these four severe cases, more than half of the glomerular segmental sclerosis area was localized in the perihilar region. The average number of ADAMTS13-positive cells per glomerulus was significantly lower in USS cases than controls (p < 0.05). C4d staining was significantly more prevalent in the glomerular capillary walls of USS cases than controls (p < 0.05).
Results date posted
2020 Year 11 Month 25 Day
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics 
Upshaw-Schulman syndrome (USS) is a congenital form of thrombotic thrombocytopenic purpura (TTP) associated with loss-of-function mutations in the ADAMTS13 gene, possibly leading to aberrant complement activation and vascular injury. However, USS is extremely rare, and there have been no systematic studies correlating histopathological severity with local ADAMTS13 expression and complement activation.
Participant flow 
We compared histopathological features, ADAMTS13 immunoreactivity, and immunoreactivity of complement proteins C4d and C5b-9 among renal biopsy tissues from five USS cases, ten acquired TTP cases, and eleven controls.
Adverse events 
None
Outcome measures 
Immunohistochemistry(ADAMTS13, C4d, C5b-9)
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status Completed
Date of protocol fixation
2017 Year 06 Month 01 Day
Date of IRB
2018 Year 03 Month 28 Day
Anticipated trial start date
2017 Year 06 Month 01 Day
Last follow-up date
2018 Year 06 Month 01 Day
Date of closure to data entry
2018 Year 06 Month 01 Day
Date trial data considered complete
2018 Year 06 Month 01 Day
Date analysis concluded
2018 Year 06 Month 01 Day

Other
Other related information Nothing in particular

Management information
Registered date
2017 Year 05 Month 24 Day
Last modified on
2020 Year 11 Month 25 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000031482