Duchenne muscular dystrophy patients over 10 years old
Conditions
Interventions
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 adjus
Sponsors
Matsumura Tsuyoshi
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion 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%, and desaturation index<=4% in sleep study (6) Patients with LVEF of 30% or more (7) Patients with written consent or assent from the patient or the guardian
Exclusion criteria
Exclusion 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 undergone gene transfer therapy (including clinical trials); who have participated in other clinical trials or therapeutic intervention trials within 6 months prior to the start of administration of the investigational drug, or are scheduled to participate in other clinical trials or therapeutic intervention studies during the study period; patients scheduled to participate in other clinical trials or therapeutic intervention trials during the study period. (6) Patients who have undergone exon skipping therapy within 6 months prior to administration of the investigational drug. (7) Patients who have had or are scheduled to undergo spinal correction surgery (8) Patients with severe renal dysfunction (eGFR <30mL/min/1.73m2 due to cystatin C) (9) 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) (10) Patients with marked white blood cell (WBC) decrease (less than 3,000/mm3) and platelet (Plt.) decrease (less than 75,000/mm3) (11) Patients with drinking habits (12) Patients with a history of smoking (13) Patients who have used Tranilast within 6 months before obtaining consent (14) Patients with a history of hypersensitivity to Tranilast (15) Judged inappropriate to participate in this study by the principal investigator or sub-investigator (16) Female patients
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The total score of PUL ver. 2.0 %FVC, %PEF LVEF | — |
Secondary
| Measure | Time frame |
|---|---|
| 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 | — |
Contacts
Public ContactTsuyoshi Matsumura
NHO Osaka Toneyama Medical Center
Outcome results
None listed