Givinostat Phase 3 Trial Recruiting

 

A phase 3 clinical trial of the investigational drug Givinostat is now recruiting in the USA, Canada and Europe.  The main purpose of the Phase 3 trial is to determine if Givinostat is able to slow the disease progression in children with Duchenne muscular dystrophy. The study will also assess safety and tolerability of the drug. Givinostat is being developed by Italfarmaco.

Givinostat is an HDAC inhibitor that has the potential to benefit all individuals with Duchenne regardless of their dystrophin Glossary Link gene Glossary Link mutation.

HDAC inhibitors have been shown to stimulate muscle growth and counter muscle degeneration in preclinical studies in the mdx mice and in a Phase 2 clinical trial in Duchenne boys. The Phase 2 trial is ongoing in Europe, and after 12 months of treatment with Givinostat the boys had significantly increased muscle fiber size, significantly decreased total fibrosis, and significantly increased regenerative fibers.  The functional tests showed an overall stability after 12 months of treatment and the drug was safe and well tolerated.  

In order to enroll in this phase 3 trial, participants must be:

  • Duchenne boys from 6 years of age
  • Ambulatory
  • Able to perform the 4 stair climb in 8 seconds or less
  • On a stable dose of corticosteroid for at least 6 months prior to start the treatment

Additional details regarding inclusion and exclusion criteria are available on ClinicalTrials.gov (NCT#02851797).

Approximately 200 boys will be recruited into this study.  Each participant will be randomized to receive either Givinostat oral suspension (10 mg/mL) twice daily OR placebo.  The ratio of participants on Givinostat versus placebo is 2:1.  The outcome measures for this study include the 4 stair climb, the 6 minute walk test and time to rise from floor.  MRI/MRS (Magnetic Resonance Imaging/ Spectroscopy) will also be performed. 

This study will have about 15 sites in the USA, 3 sites in Canada and about 23 sites in Europe.  Reasonable travel and accommodations will be provided or reimbursed.  If you wish to participate or have questions, please visit ClinicalTrials.gov (NCT#02851797) for contact information for each trial site.

You may also email This email address is being protected from spambots. You need JavaScript enabled to view it.

In addition, please view Italfarmaco webinar hosted by Parent Project Muscular Dystrophy on January 24, 2018 for a closer look into this trial.

Posted 2/2/2018