Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy, safety and tolerability of givinostat in non-ambulant patients with Duchenne Muscular Dystrophy

Conditions

Duchenne muscular dystrophy (DMD)

Brief summary

Change of PUL total score at 18 months of treatment of givinostat compared to placebo group

Detailed description

•Change from baseline of Peak Expiratory Flow percent predicted (PEF%p) at 18 months of treatment of givinostat compared to placebo group • Change from baseline of Forced Vital Capacity percent predicted (FVC%p) at 18 months of treatment of givinostat compared to placebo group • Cumulative loss of PUL items over 18 months of treatment of givinostat compared to placebo group, •Type incidence and severity of TEAEs •Proportion of patients experiencing TEAEs (baseline to EOS/F-Up) • Changes in vital signs, lab tests, ECG, ECHO •Time to assisted ventilation, rate and severity of respiratory infection and duration and use of antibiotics of givinostat to placebo group •Time to onset of diarrhoea •Height and weight Z-scores

Related papers

No related papers found yet.

Interventions

DRUGPLACEBO for GIVINOSTAT hydrochloride monohydrate (ITF2357) 10 mg/mL oral suspension

DRUGITF2357

Eligibility

Sex/Gender

Male

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Change of PUL total score at 18 months of treatment of givinostat compared to placebo group	—

Secondary

Measure	Time frame
•Change from baseline of Peak Expiratory Flow percent predicted (PEF%p) at 18 months of treatment of givinostat compared to placebo group • Change from baseline of Forced Vital Capacity percent predicted (FVC%p) at 18 months of treatment of givinostat compared to placebo group • Cumulative loss of PUL items over 18 months of treatment of givinostat compared to placebo group, •Type incidence and severity of TEAEs •Proportion of patients experiencing TEAEs (baseline to EOS/F-Up) • Changes in vital signs, lab tests, ECG, ECHO •Time to assisted ventilation, rate and severity of respiratory infection and duration and use of antibiotics of givinostat to placebo group •Time to onset of diarrhoea •Height and weight Z-scores	—

Measure

Time frame

•Change from baseline of Peak Expiratory Flow percent predicted (PEF%p) at 18 months of treatment of givinostat compared to placebo group • Change from baseline of Forced Vital Capacity percent predicted (FVC%p) at 18 months of treatment of givinostat compared to placebo group • Cumulative loss of PUL items over 18 months of treatment of givinostat compared to placebo group, •Type incidence and severity of TEAEs •Proportion of patients experiencing TEAEs (baseline to EOS/F-Up) • Changes in vital signs, lab tests, ECG, ECHO •Time to assisted ventilation, rate and severity of respiratory infection and duration and use of antibiotics of givinostat to placebo group •Time to onset of diarrhoea •Height and weight Z-scores

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Countries

Belgium, France, Germany, Italy, Netherlands

Outcome results

None listed