Upper Limb performance in non-ambulatorY patientS on givinoStat and stEroidS (ULYSSES)

• Research type

  Research Study

• Full title

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

• IRAS ID

  1008441

• Contact name

  Serena Franciosi

• Contact email

  s.franciosi@italfarmacogroup.com

• Sponsor organisation

  Italfarmaco SpA

• Eudract number

  2023-503521-19

• Clinicaltrials.gov Identifier

  NCT05933057

• Research summary

  Duchenne muscular dystrophy (DMD) is a rare condition which affects the muscles and leads to muscle wasting. This is due to the changes of a protein in your body called dystrophin. Dystrophin helps keep muscle cells healthy. DMD usual affects boys but can occasionally affect girls, although this is rare and usually milder. The usual treatment for DMD includes a medicine which can have bad side effects. Givinostat treats DMD by helping to protect the muscles and help them repair.
  The study is for boys aged 9 to 17 who have DMD and are unable to walk unaided. The study will test how well Givinostat works to help protect and repair the muscles, how safe the medicine is and what the side effects are like.
  The study will involve taking the medicine for 18 months and participants will have regular (up to 14) visit at their hospital to see how they are getting on.
  Each participant will be randomly assigned (like rolling a dice) to either receive Givinostat or placebo. Both Givinostat and placebo will be given as a solution (like a syrup) that is taken by mouth. A ‘placebo’ looks like Givinostat but does not have any of the active medicine in it. Each participant will have a 2 in 3 chance of getting Givinostat and a 1 in 3 chance of getting placebo. This is a ‘blinded’ study which means that neither the participants nor the study doctors will not know which treatment is receiving. This is the best way to measure the effect of Givinostat.

• REC name

  HSC REC B

• REC reference

  23/NI/0118

• Date of REC Opinion

  16 Oct 2023

• REC opinion

  Further Information Favourable Opinion