Testing Givinostat against a placebo for duchenne muscular dystrophy

Exclusion

• −Hypersensitivity to any component of study medication
• −Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screeningScreeningThe checks done before joining, to see whether a study fits.Read more →
• −Patients will be excluded from the study if they satisfy any of the following criteria:
• −Exposure to another investigational drug within 3 months prior to start of study treatment.
• −Have exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study treatment
• −Having received any gene therapy (eg, AAV Micro-dystrophin delivery) prior to start of study treatment
• −Use of any pharmacologic treatment or supplement (other than corticosteroids), that might have had an effect on muscle strength or function within 3 months prior to the start of study treatment (eg, growth hormone); vitamin D, calcium and any other supplements will be allowed
• −Use of testosterone, unless used as a replacement therapy for the treatment of delayed puberty. The testosterone dose and regimen should be stable within 6 months prior to the start of study treatment, and circulating testosterone levels should be within the normal ranges for the patient's age
• −Elbow-flexion contractures \>30° in the dominant armArmOne of the groups in a study, each receiving something different.Read more →
• −Inability to perform consistent PUL 2.0 measurement within ±2 points without shoulder domain or within ±3 points with shoulder domain during paired testing at screening
• −Forced Vital Capacity % of predicted \<40%
• −Requirement for daytime ventilator assistance (Note: Night ventilator assistance and use of bi-level positive airway pressure therapy is allowed)
• −Episode of respiratory failure within the 8 weeks prior to screening
• −Symptomatic cardiomyopathy or heart failure and/or left ventricular ejection fraction \<45%
• −BaselineBaselineYour starting measurements, taken before treatment begins.Read more → corrected QT interval using Fredericia's formula (QTcF) \>450 msec (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (eg, heart failure, hypokalaemia, or family history of long QT syndrome)
• −Poorly controlled asthma or underlying lung disease such as bronchitis, bronchiectasis, emphysema, recurrent pneumonia that in the opinion of the InvestigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more → might impact respiratory function
• −Platelets, white blood cells, and/or haemoglobin \< lower limit of normal (LLN) at screening (Note: for abnormal screening laboratory test results \[\<LLN\], the platelets count, white blood cell, and haemoglobin will be repeated once; if the repeat test result is still \<LLN, the patient should be excluded)
• −Fasting triglycerides \>300 mg/dL (3.42 mmol/L) at screening (Note: if the value is \>300 mg/dL, the triglycerides will be repeated once; if the repeated test result is still \>300 mg/dL, the patient should be excluded)
• −Current or history of liver disease or impairment, including but not limited to a baseline elevated total bilirubin (ie, \>1.5 × upper limit of normal \[ULN\]), unless secondary to Gilbert disease or pattern consistent with Gilbert disease
• −Inadequate renal function, as defined by serum Cystatin C result \>2 × ULN (Note: if the value is \>2 × ULN, the serum Cystatin C will be repeated once; if the repeated test result is still \>2 × ULN, the patient should be excluded)
• −Sorbitol intolerance or malabsorption, or have the hereditary form of fructose intolerance
• −Diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD, based on Investigator judgement
• −Psychiatric illness or social situations rendering the potential patient unable to understand and comply with the muscle function tests and/or with the study protocolProtocolThe detailed plan a study must follow.Read more → procedures, based on Investigator judgement
• −Have contraindications to MRI scan (eg, claustrophobia, metal implants, or uncontrolled seizure disorder), based on Investigator's judgement.
• −Major surgical procedure (including scoliosis surgery) planned within 1 year of the start of study treatment