Givinostat

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

Questions and Answers

What stage is this research?

This Phase 3 trial is actively recruiting participants in US, Canada, and Europe.

What is the goal or purpose of this study?

The main goal of this phase 3 study is to evaluate the efficacy of Givinostat compared to placebo to slow the disease progression in Duchenne boys. The study will also assess safety and tolerability of the drug.

Has Givinostat been tested before?

Givinostat has been tested in a number of clinical studies in adults and pediatric populations. Concerning the clinical experience in dystrophinopathies, a Phase 2 clinical trial (DSC/11/2357/43) has been conducted to evaluate the safety and the potential Givinostat as a treatment for Duchenne.
DSC/11/2357/43 was an open label 2-part, Phase 2 study in ambulatory children aged ≥ 7 to less than 11. Clinical results showed that Givinostat significantly counteracted histological disease progression after 12 months of treatment, in particular:

  • It significantly increased muscle fibers size, and it significantly decreased total fibrosis, fatty replacement, necrosis, the mean number of hypercontracted fibers;
  • It significantly increased regenerative fibers with no depletion of the pool of satellite cells.

The functional tests showed an overall stability after 12 months of treatment in this population, with small changes in some parameters. Overall the drug was safe and well tolerated. The study was completed in December 2017, 18 boys are still on treatment in the long-term safety study (DSC/14/2357/51). The results of this Phase 2 study, 12 months of treatment were published in Neuromuscular Disorders, in October 2016 (Bettica et al, 26(10):643-649).

Who is funding this study?

This study is sponsored by Italfarmaco SpA.

Who is eligible to participate in this study?

Clinicians are recruiting approximately 192 ambulant Duchenne boys. Of these, 128 subjects will be assigned to Givinostat and 64 will be assigned to placebo. To participate in this study you must be:

  • Male with Duchenne diagnosis confirmed by genetic testing
  • Age ≥6 years
  • On stable corticosteroid for at least 6 months
  • Able to perform the 4 stairs climb in not more than 8 seconds
  • Able to rise from floor in less than 10 seconds
  • Additional details regarding inclusion and exclusion criteria will be available on
  • www.ClinicalTrials.gov (NCT#02851797).

What do I have to do if I decide to participate in this study?

This study involves clinic visit every 12 weeks for 18 months, with more frequent visits during the first 3 months. After the screening period, boys will be randomized to receive Givinostat oral suspension or placebo oral suspension to be taken twice a day with food (e.g. during breakfast and during dinner).

How long will this study last, and will I have access to the drug/treatment once the study has ended?

The anticipated length of the study is 18 months of double-blind, placebo controlled period. When a subject has concluded 18 months of treatment (Givinostat or Placebo), regardless his walking ability, he will be asked to participate in an open-label long-term safety study with Givinostat, until the drug will be available on the market. All subjects in this long-term safety study (DSC/14/2357/51, NCT#03373968 ) will take Givinostat.

Where does this study take place?

This study takes place at many sites in US, Canada, and Europe.

  • How many visits to the study site are necessary?
  • Approximately 15 visits over the 18 months duration of the study. During the screening period, the patient will have 2 visits, and he will be asked to come back to the site for an additional visit to have an MRI scan which could be done in a different hospital. Then, he will come back within 1 month for the first administration of the study treatment. During the first month of treatment the patients will come back to the site weekly, then every 2 weeks for the second months, then at the third months and then every 3 months.
      • Can any visits be done locally?
    • Yes, a study nurse is appointed to collect blood sample at your home (if you agree) in some visits
    • Is there any funding to help pay for travel?
    • Yes, there is a reimbursement for the travel
    • .

Will I get paid for participating in this study?

No, you won’t. Costs associated with participation in the study (e.g. travel, parking, hotel, meals) are reimbursed. The reimbursement process is different from countries, in some countries Italfarmaco through the hospital will reimburse your travel expenses incurred for the participation of the study, in other countries (e.g. US and Canada) a travel agency can be involved to plan your travel and reimburse the travel expenses.

Why should I consider participating in this study?

While no personal benefit can ever be guaranteed by participation in a clinical trial, there are other benefits, including allowing you to play an active role in your own health care (or that of your child), gaining access to new research treatments before they are widely available and having access to medical specialists that are normally not available to you or your child, and helping others by contributing to the better understanding of Duchenne.

Where can I learn more about this study?

You can learn more about givinostat studies at www.italfarmaco.com and www.ClinicalTrials.gov (Epidys trial NCT#02851797, long-term trial NCT#03373968 and Becker trial NCT#03238235).
You may contact clinical study staff to inquire about clinical study.

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