Randomized, double-blind, placebo-controlled Phase II study to evaluate the efficacy and safety of Tranilast on motor, respiratory, and cardiac function in patients with Duchenne muscular dystrophy aged 10 years and older

JRCT ID: jRCT2051230201

Registered date:12/03/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Duchenne muscular dystrophy patients over 10 years old
• Date of first enrollment: 01/04/2024
• Target sample size: 30
• Study type: Interventional
• Intervention(s): During the 52-weeks implementation period, the active drug group will receive Tranilast 100mg x 3 times/day orally. For patients younger than 15 years of age at the time of treatment, it will be adjusted to 5 mg/kg/day in 3 divided doses. The placebo group will receive the placebo orally 3 times/day.

Outcome(s)

Primary Outcome

The total score of PUL ver. 2.0
%FVC, %PEF
LVEF

Secondary Outcome

Grip strength in the 9HPT
Serum CK value
FVC, PEF, AHI in the sleep study, desaturation index, mean SpO2
NT-proBNP, cTnI, LVDd, LVFS
TRPV2 expression on the surface of peripheral blood mononuclear cells
EQ-5D, DMDSAT
Introduction of artificial respiration therapy, cardiac events, all-cause mortality
Adverse event

Key inclusion & exclusion criteria

• Age minimum: >= 10age old
• Age maximum: Not applicable
• Gender: Male

Include criteria

(1) Patients aged 10 years or older at the time of consent
(2) Patients diagnosed with Duchenne muscukat dystropht (genetic, immunological diagnosis)
(3) Patients who can take the investigational drug orally
(4) Patients with an entry item of 1 or more in the Performance of upper limb, ver. 2.0
(5) Patients with sufficient respiratory funtion who meet all of the following conditions:
%FVCH>=0%, desaturation index<=4%, and AHI<=10 in sleep study
(6) Patients with LVEF of 30% or more
(7) Patients with written consent or assent from the patient or the guardian

Exclude criteria

(1) Patients undergoing artificial respiration therapy
(2) Patients whose steroid prescription has been changed within 3 months for reasons other than adjusting the dose due to weight change
(3) Patients whose dose of heart failure drugs has been changed within 3 months
(4) Patients with acute heart failure
(5) Patients who have participated in clinical trials at the gene/protein level such as gene transfer therapy, exon skipping therapy, and read-through therapy; who have participated in other clinical trials and therapeutic intervention trials within 1 year before the start of administration of the investigational drug; patients scheduled to participate in other clinical trials or therapeutic intervention trials during the study period.
(6) Patients who have had or are scheduled to undergo spinal correction surgery
(7) Patients with severe renal dysfunction (eGFR <30mL/min/1.73m2 due to cystatin C)
(8) Patients with severe liver dysfunction (T.Bil. is 1.5 times or more than the normal upper limit; AST, ALT, ALP is 5 times or more than that, only if the effects of skeletal muscle degeneration or age can be ruled out; PT is 40% or less; having bleeding tendency, liver failure (disturbance of consciousness, fulminant hepatitis), liver cirrhosis, liver tumor, jaundice that lasts for 6 months or more (equivalent to grade 3 in the Classification Criteria for Severity of Adverse Drug Reactions)
(9) Patients with marked white blood cell (WBC) decrease (less than 3,000/mm3) and platelet (Plt.) decrease (less than 75,000/mm3)
(10) Patients with drinking habits
(11) Patients with a history of smoking
(12) Patients who have used Tranilast within 6 months before obtaining consent
(13) Patients with a history of hypersensitivity to Tranilast
(14) Judged inappropriate to participate in this study by the principal investigator or sub-investigator
(15) Female patients

Related Information

• Primary Sponsor: Matsumura Tsuyoshi
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development