Givinostat/Placebo/DMD-Blinded Phase 3

Clinical Trial

Offered by: Nemours Children's
Location: Orlando, Fla.

Trial Name

Givinostat vs. Placebo/Duchenne Muscular Dystrophy — Phase 3-Italfarmaco Protocol #DSC/14/2357/48

What is the trial about?

This is a blind study to learn how well a medicine called Givinostat (the study drug) works for boys with Duchenne Muscular Dystrophy (DMD), and to see how safe Givinostat is compared to a placebo (a placebo looks like the study drug but does not contain any active study drug).

Who can participate?

Ambulant males aged ≥ 6 years with DMD signs or symptoms; DMD diagnosis confirmed by genetic testing; able to complete two (2) Four Stairs Climb test; taking a stable dose of steroids for minimum of six months.  

What is involved?

The length of the study is approximately 20 months (about two years). The study is divided into two time periods: a screening period (which will start about four weeks before the first dose of study drug) and a treatment period (which will include 18 months of treatment with the study drug).

Study Visits: 15 study visits through the treatment period (three of the study visits may be completed at participant's home or at regular doctor’s office). At the end of the study (about one month after the last dose of study drug), if participant does not continue in the long-term follow-up study, there will be a final follow-up visit.  

At the end of the 18-month treatment period of this study, there will be the option to participate in a long-term study.

• Physical examination to be done 12 times (visits 1, 3, 4, 7, 9-16)
• Measurement of blood pressure, heart rate and temperature to be done 12 times (visits 1, 3, 4, 7, 9-16)
• Electrocardiogram (ECG — heart test) to be done 12 times (visits 1, 3, 4, 7, 9-16)
• Echocardiogram (ECHO) to be done twice (visits 1 and 15)
• Pulmonary function tests (breathing tests) to be done 3 times (visits 1, 13 and 15)
• Muscular strength assessments to be done 9 times (visits 1, 2, 3, 10-15)
• Blood samples to be drawn 15 times (All visits except visit 2); urine sample to be collected 9 times (visits 1, 9-16)
• Questionnaire (for parent) to be completed 3 times (visits 1, 13 and 15); cognitive thinking test for child to be completed twice (visits 1 and 15)
• Rate the taste of the study drug to be done twice (visits 7 and 15)
• Magnetic Resonance Imaging (MRI)/Magnetic Resonance Spectroscopy (MRS) test, only if applicable) to be done 3 times (visits 2, 13 and 15)