Phase 3 study of Givinostat in people with Duchenne

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 trial is to evaluate the efficacy of Givinostat compared to placebo to slow the disease progression in boys with Duchenne muscular dystrophy. 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 adult and pediatric populations with cancer and arthritis. We completed a Phase 2 clinical trial (DSC/11/2357/43) to evaluate the safety and the potential efficacy of Givinostat among boys with Duchenne.

DSC/11/2357/43 was an open label, 2-part, Phase 2 trial in ambulatory boys with Duchenne who were 7 years up to 11 years old. Trial results showed that Givinostat significantly reduced histological disease progression after 12 months of treatment, in particular:

It significantly increased the size of muscle fibers, 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.

Analysis of functional testing found that participants’ muscle strength and function were stable after 12 months of treatment, 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 a long-term safety study (DSC/14/2357/51). The results of the 12-month Phase 2 trial 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 224 ambulatory boys with Duchenne. Of these, 160 participants will be randomly assigned to Givinostat and 80 will be randomly assigned to placebo. To participate in this study you must be:
 Male with a Duchenne diagnosis confirmed by genetic testing
 Age ≥6 years
 On a stable corticosteroid for at least 6 months
 Able to perform the 4 stair climb in not more than 8 seconds
 Able to rise from the floor in 3 up to 10 seconds
 There are other inclusion and exclusion criteria for this study. You can find all of the details on www.ClinicalTrials.gov (NCT#02851797).

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

 This study involves 15 clinic visits during an 18-month period. Visits are more frequent during the first 3 months. After the screening period, boys will be randomly assigned to receive Givinostat oral suspension or placebo oral suspension taken twice a day with food (e.g. during breakfast and dinner).
 Trial visits include physical function testing, blood tests, urine tests, and physical exams.
 Participants also complete three MRI tests to measure changes inside the muscle.

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 trial is 18 months. Participants who finish the double – blind placebo – controlled study can enroll in an open-label long-term safety study to receive Givinostat oral liquid suspension until the drug becomes available on the market. Participants who finish the 18-month study are eligible for the open label study regardless of their walking or stair climbing ability. All participants in this long-term safety study (DSC/14/2357/51, NCT#03373968) will take Givinostat oral liquid suspension.

Where does this study take place?

 This trial takes place at many sites in US, Canada, and Europe.
 How many visits to the trial site are necessary?
• Trial participants complete approximately 15 visits over 18 months. During the screening period, boys attend two visits. Once those are finished, boys have an MRI test, which might be at another site. Then boys return for one more visit to receive the first dose of the study drug. During the first month of treatment, boys attend weekly visits, then every two weeks in the second month, then once in the third month, and then every three months.
 Can any visits be done locally?
• Yes, a trial nurse is appointed to collect blood sample at your home (if you agree) in some visits. This service is optional.
 Is there any funding to help pay for travel?
• Yes. In the US and Canada, a travel agency will arrange your travel for you. In other countries, you will be reimbursed for your travel costs. Ask your site for more details.

Will I get paid for participating in this study?

 No, you won’t. Costs associated with participation in the trial (e.g. travel, parking, hotel, meals) are reimbursed. The reimbursement process is different in every country. In some countries Italfarmaco will arrange for your expenses to be reimbursed by your site. In other countries (US and Canada), we offer central travel support through a travel agency.

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 can email us at patientadvocacy@italfarmaco.com to ask more questions.

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