Tranilast-MD

• Register: JPRN
• Last refreshed on: 17 October 2023
• Main ID: JPRN-jRCTs031180038
• Date of registration: 12/11/2018
• Prospective Registration: Yes
• Primary sponsor: Matsumura Tsuyoshi
• Public title: Tranilast-MD
• Scientific title: A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy - Tranilast-MD
• Date of first enrolment: 18/12/2018
• Target sample size: 20
• Recruitment status: Not Recruiting
• URL: https://jrct.niph.go.jp/latest-detail/jRCTs031180038
• Study type: Interventional
• Study design: single arm study, open(masking not used), no treatment control/standard of care control, single assignment, treatment purpose
• Phase: N/A

Contacts

• Name: Tsuyoshi Matsumura
• Affiliation: National Hospital Organization OsakaToneyama Medical Center

• Name: Yutaka Ito
• Affiliation: National Hospital Organization Nagoya Medical Center

Key inclusion & exclusion criteria

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
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

Age minimum: >= 13age old
Age maximum: Not applicable
Gender: Both

Health Condition(s) or Problem(s) studied

muscular dystrophy ,heart failure ,tranilast

muscular dystrophy
muscular dystrophy ,heart failure ,tranilast

muscular dystrophies

Intervention(s)

Tranilast 300 mg / day is administered three times per minute.
Treatment for 28 weeks (in principle, outpatient administration). As of the 28th week, reconfirmation of consent regarding continuation of administration is confirmed, and if confirmation is obtained, further treatment for 116 weeks is carried out.

Primary Outcome(s)

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)

Secondary Outcome(s)

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

Secondary ID(s)

UMIN000031965

Source(s) of Monetary Support

National Hospital Organization

Ethics review

Status: Approval
Approval date: 23/10/2018
Contact:

[e-mail]

National Hospital Organization Review Board for Clinical Trials (Nagoya)

[phone]

[e-mail]