UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000031965
Receipt number R000036486
Scientific Title A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy
Date of disclosure of the study information 2018/03/30
Last modified on 2020/11/20 16:29:49

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Basic information

Public title

A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy

Acronym

Tranilast-MD

Scientific Title

A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy

Scientific Title:Acronym

Tranilast-MD

Region

Japan


Condition

Condition

Heart failure patients with muscular dystrophy who showed brain natriuretic peptide (BNP) of 100 pg/mL or more in spite of myocardial protection treatment

Classification by specialty

Cardiology Neurology

Classification by malignancy

Others

Genomic information

NO


Objectives

Narrative objectives1

To assess the safety and efficacy, such as decrease of brain natriuretic peptide (BNP)/cardiac events and improvement of cardiac function, by using tranilast in combination with other drugs in heart failure patients with muscular dystrophy who showed BNP of 100 pg/mL or more in spite of myocardial protection treatment in an open-label, single-arm study.

Basic objectives2

Safety,Efficacy

Basic objectives -Others


Trial characteristics_1

Exploratory

Trial characteristics_2


Developmental phase



Assessment

Primary outcomes

The change in BNP before the start of administration (using the average of values in the pre-treatment observation period and at the start of administration) to 24 weeks (using the average of values at 20 weeks, 24 weeks and 28 weeks)

Key secondary outcomes

1) Cardiac events (change of oral medicine for cardiac failure due to cardiac function exacerbation (ACEI/ARB, Beta blocker, digitalis, diuretic, aldosterone antagonist, cardiotonic agent or antiarrhythmic agent), administration of intravenous drugs (cardiotonic agents, diuretics or antiarrhythmic agent), hospitalization due to heart failure or prolongation of hospitalization)
2) All deaths
3) Left ventricula fractional shortening (FS)
4) Human atrial natriuretic peptide (hANP), cardiac troponin T (cTnT)
5) The expression of transient receptor potential cation channel, subfamily V, member 2 (TRPV2) expression on cytoplasminc membrane of isolated peripheral blood mononuclear cells (PBMCs)
6) Hand finger muscle strength (pinch strength), creatine kinase (CK)
7) Muscular dystrophy quality of life-60 (MDQOL-60), The short form (12) health survey (SF-12)
8) Adverse events


Base

Study type

Interventional


Study design

Basic design

Single arm

Randomization

Non-randomized

Randomization unit


Blinding

Open -no one is blinded

Control

Uncontrolled

Stratification


Dynamic allocation


Institution consideration


Blocking


Concealment



Intervention

No. of arms

1

Purpose of intervention

Treatment

Type of intervention

Medicine

Interventions/Control_1

Tranilast 300 mg/day is administered three times per day as the study treatment.
The start date of study treatment is defined as the medication start date, and 28 weeks of study treatment will be performed (in principle, by outpatient administration). We reconfirm consent for continuation of administration at 28 weeks, and if consent is obtained, further treatment for 116 weeks will be carried out.
As long as there are no particular problems, the outpatient visit during the observation period is in 4 weeks (21-35 days), 12 weeks (77-91 days), 20 weeks (134-147 days), 24 weeks (161-175 days), and 28 weeks (190-203 days) after starting medication. A prescription until the next outpatient visit will be issued at the consultation. The outpatient visit after consent reconfirmation is done at 12-weeks intervals after 36 weeks, and the prescription until the next outpatient visit will be issued until the next 144 weeks after starting medication at the consultation.
Evaluation of various examination findings including clinical findings, cardiac functions, respiratory functions, motor function, QOL Questionnaire (MDQoL-60, SF-12), and adverse events at the designated timing.
In order to confirm TRPV2 inhibitory effect by tranilast and to assess its effectiveness as a biomarker, central laboratory tests for the TRPV2 expression analysis will be performed.

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

13 years-old <=

Age-upper limit


Not applicable

Gender

Male and Female

Key inclusion criteria

1) MD patients aged 13 or more
2) With high value in BNP (100 pg/mL or more)
3) Those introduced with standard myocardium protective drugs (angiotensin converting enzyme inhibitor (ACEI)/angiotensin type II receptor blocker (ARB) and/or beta blocker) who meets both of the following:
taking maintenance doses at the time of consent;
whose dosage regimen and doses are fixed from 2 weeks before the start of administration until the start of administration.
4) To whom intrinsic administration of capsule, fine granules or dry syrup is possible, or who can be reliably administered tranilast by tube
5) Provided written consent by their free will/the representative

Key exclusion criteria

1) Acute stage heart failure condition (using cardiotonic, diuretic, antiarrhythmic drug intravenously)
2) From 2 weeks before the start of administration to the start of administration Directions of digitalis, diuretic, aldosterone antagonist, cardiotonic agent, antiarrhythmic drug are not fixed
3) With a lethal arrhythmia including ventricular premature contraction of more than four (short run)), excluding those with transplanted implantable defibrillators
4) With serious renal dysfunction (estimated glomerular filtration ratio (eGFR) using cystatin C of less than 30 mL/min/1.73 m2)
Male: eGFR = (104 ^ Cystatin C-1.019^ 0.996age (years)) - 8
Female: eGFR = (104 ^ Cystatin C-1.019 ^ 0.996age (years) ^ 0.929) - 8
For those aged 18 or less, cyctain C of 2.5 mg/L or more is used.
5) With severe liver function disorder (T. Bil of 10 mg/dl or more, AST and ALT of 500 IU/L or more, ALP of 5 times or more of the normal upper limit, PT of 40% or less, bleeding tendency, hepatic failure symptoms (fulminant hepatitis), cirrhosis of the liver, liver tumor, jaundice prolonged for more than 6 months) (equivalent to grade 3 in "Classification criteria for severity of adverse drug reactions" )
6) Marked white blood cell (WBC) decrease (less than 3000/mm^3), platelet (Plt) decrease (less than 80,000/mm^3)
7) Having a history of hypersensitivity to tranilast
8) Pregnant or possibly pregnant
9) For whom the principal investigator/sub-investigators judged not appropriate for participation in this study

Target sample size

20


Research contact person

Name of lead principal investigator

1st name
Middle name
Last name Tsuyoshi Matsumura

Organization

National Hospital Organization Toneyama National Hospital

Division name

Neurology

Zip code


Address

5-1-1 Toneyama, Toyonaka, Osaka 560-8552 Japan

TEL

06-6853-2001

Email

tmatsumura-toneyama@umin.org


Public contact

Name of contact person

1st name
Middle name
Last name Yutaka Ito

Organization

National Hospital Organization Nagoya Medical Center

Division name

Clinical Research Center

Zip code


Address

4-1-1 Sannnomaru Naka-ku, Nagoya 460-001, Japan

TEL

052-951-1111

Homepage URL


Email

study.office@nnh.go.jp


Sponsor or person

Institute

National Hospital Organization Toneyama National Hospital

Institute

Department

Personal name



Funding Source

Organization

National Hospital Organization

Organization

Division

Category of Funding Organization

Other

Nationality of Funding Organization

Japan


Other related organizations

Co-sponsor


Name of secondary funder(s)



IRB Contact (For public release)

Organization


Address


Tel


Email



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

2018 Year 03 Month 30 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

2017 Year 12 Month 04 Day

Date of IRB

2019 Year 10 Month 23 Day

Anticipated trial start date

2018 Year 12 Month 14 Day

Last follow-up date

2023 Year 03 Month 31 Day

Date of closure to data entry


Date trial data considered complete


Date analysis concluded

2023 Year 03 Month 31 Day


Other

Other related information



Management information

Registered date

2018 Year 03 Month 29 Day

Last modified on

2020 Year 11 Month 20 Day



Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000036486


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name