UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000027482
Receipt number 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 09:40:19

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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 or person

Institute

Nara medical university

Institute

Department

Personal name



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

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000031482


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name