Researchers at Italfarmaco are looking for participants with Duchenne muscular dystrophy (DMD) to participate in a phase 3 study. The goal of the study is to evaluate the efficacy and safety of givinostat, a drug that belongs to a class called HDAC inhibitors and is believed to induce the muscle repair process, which may prevent or repair muscle damage in patients with DMD.
The study will determine whether treatment with givinostat is associated with stabilized or slowed disease progression as measured by change in the time to climb four stairs.
Participants will be randomized to receive either placebo or givinostat, with an option to receive the study drug during an open-label extension period. The trial will consist of approximately 16 clinic visits that will include a series of vital and functional tests.
To be eligible to participate, individuals must:
- Be male, older than 6 years
- Be diagnosed with genetically confirmed DMD
- Be receiving systemic corticosteroids for a minimum of six months (with no significant change in dosage or dosing regimen for a minimum of six months) prior to the start of the study
- Be able to climb four stairs in £ eight seconds (± one second on two trials)
- Be able to rise from the floor between ³ three and < 10 seconds
- Have a manual muscle testing (MMT) of quadriceps grade ³ three
- Meet additional study criteria
Travel support is available.
To learn more or to inquire about participation, contact email@example.com or call 412-593-4389.
To find out more information about the study, you may also visit https://www.clinicaltrials.gov/ct2/show/NCT02851797 or the Italfarmaco website at https://www.italfarmaco.com/en-us/Activities/Pipeline/Clinical-trials/Givinostat-for-DMD.