UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000044637
Receipt number R000050987
Scientific Title Development of diagnostic and disease-monitoring biomarkers of bradykinin-mediated angioedema with or without C1-inhibitor deficiency in hereditary angioedema
Date of disclosure of the study information 2021/08/06
Last modified on 2023/12/13 09:03:59

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Basic information

Public title

Development of diagnostic and disease-monitoring biomarkers of bradykinin-mediated angioedema with or without C1-inhibitor deficiency in hereditary angioedema

Acronym

HAE BMA study

Scientific Title

Development of diagnostic and disease-monitoring biomarkers of bradykinin-mediated angioedema with or without C1-inhibitor deficiency in hereditary angioedema

Scientific Title:Acronym

HAE BMA study

Region

Japan


Condition

Condition

Hereditary angioedema

Classification by specialty

Medicine in general Hematology and clinical oncology Nephrology
Clinical immunology Dermatology Adult
Child

Classification by malignancy

Others

Genomic information

NO


Objectives

Narrative objectives1

This observational study will be conducted to investigate the usefulness of candidate blood biomarkers in the plasma of HAE patients as novel biomarkers for HAE by quantification and activity measurements, using the proportion of cleaved HMWK (cHMWK) as a reference indicator.

Basic objectives2

Others

Basic objectives -Others

NA

Trial characteristics_1

Exploratory

Trial characteristics_2

Others

Developmental phase

Not applicable


Assessment

Primary outcomes

Evaluation of the following potential candidate blood biomarkers, including investigation of correlations between the candidate biomarkers and cHMWK will be performed.

-Kallikrein activity
-Sgp120 fragments
-PAI-2
-C1-INH activity (using dried blood spot (DBS))

Key secondary outcomes

NA


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

12 years-old <=

Age-upper limit


Not applicable

Gender

Male and Female

Key inclusion criteria

Inclusion criteria for each group shall be set as below.
Patients or individuals who meet all the criteria for the applicable group and provide consent to participate in the study or whose legally authorized representatives provide consent to participate in the study will be included.
[HAE type1/2 group]
1. Patients with symptoms due to angioedema
2. Patients with decreased C1-INH activity (50% or less)
3. Patients with a family history of HAE or a mutation of the C1-INH gene (SERPING1)
4. Patients aged 12 years or older when providing informed consent
[HAE type 3 group]
1. Patients who meet both a) and b) and were diagnosed HAE type 3
a) Have a history of recurrent angioedema without urticaria or the use of causative agents, have C1-INH activity in the range of 50% to 130%, and in whom antihistamines and steroids are ineffective for HAE attacks
b) Have a known HAE type 3 gene mutation on genetic testing or have a family history of angioedema
2. Patients aged 12 years or older when providing informed consent
[Healthy group]
1. Healthy volunteers
2. Individuals who do not have a disease (including HAE) of interest as the subject of the study
3. Individuals aged 12 years or older when providing informed consent

Key exclusion criteria

Exclusion criteria for each group shall be set as below. Patients/individuals who meet any of the criteria below for the relevant group will be excluded from the study.
[HAE type 1/2 group and HAE type 3 group]
1. Patients receiving C1-INH preparations for the 7 days prior to the time of blood collection
2. Patients receiving berotralstat hydrochloride for the 20 days prior to the time of blood collection
3. Patients receiving lanadelumab for the 70 days prior to the time of blood collection
4. Patients who are judged by the principal investigator or the subinvestigator to be inappropriate as study participants
[Healthy group]
1. Individuals who have been diagnosed with inflammatory diseases
2. Individuals who have been diagnosed with allergic diseases
3. Individuals who have not been administered a therapeutic drug continuously for 10 days or more within the 60 days* prior to the time of blood collection
4. Individuals who are judged by the principal investigator or the subinvestigator to be inappropriate as study participants

Target sample size

60


Research contact person

Name of lead principal investigator

1st name Kumiko
Middle name
Last name Hosono

Organization

Takeda Pharmaceutical Company Limited

Division name

Japan Medical Office

Zip code

103-8668

Address

1-1, Nihonbashi-Honcho 2-chome, Chuo-ku, Tokyo, Japan

TEL

080-7172-0566

Email

kumiko.hosono@takeda.com


Public contact

Name of contact person

1st name Ritsuko
Middle name
Last name Nagasaka

Organization

Takeda Pharmaceutical Company Limited

Division name

Japan Medical Office

Zip code

103-8668

Address

1-1, Nihonbashi-Honcho 2-chome, Chuo-ku, Tokyo, Japan

TEL

09014240921

Homepage URL


Email

ritsuko.nagasaka@takeda.com


Sponsor or person

Institute

Takeda Pharmaceutical Company Limited

Institute

Department

Personal name



Funding Source

Organization

None

Organization

Division

Category of Funding Organization

Self funding

Nationality of Funding Organization



Other related organizations

Co-sponsor


Name of secondary funder(s)



IRB Contact (For public release)

Organization

Kyushu University Institutional Review Board for Clinical Research

Address

3-1-1, Maidashi, Higashi-ku,Fukuoka-shi,Fukuoka

Tel

092-642-6055

Email

ijkseimei@jimu.kyushu-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

NA


Other administrative information

Date of disclosure of the study information

2021 Year 08 Month 06 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

No longer recruiting

Date of protocol fixation

2021 Year 06 Month 15 Day

Date of IRB

2021 Year 09 Month 28 Day

Anticipated trial start date

2021 Year 12 Month 07 Day

Last follow-up date

2022 Year 08 Month 31 Day

Date of closure to data entry


Date trial data considered complete

2022 Year 10 Month 14 Day

Date analysis concluded

2024 Year 01 Month 31 Day


Other

Other related information

NA


Management information

Registered date

2021 Year 06 Month 24 Day

Last modified on

2023 Year 12 Month 13 Day



Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000050987


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name