Givinostat for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

+38 other locations

Age: Any Age

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Waitlist Available

Sponsor: Italfarmaco

No Placebo Group

Prior Safety Data

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

This trial tests the safety and effectiveness of GIVINOSTAT, a liquid medicine taken by mouth, in patients with Duchenne's muscular dystrophy who have used it before. The medicine aims to improve muscle function and slow down muscle damage.

Research Team

Eligibility Criteria

This trial is for boys aged ≥6 with Duchenne Muscular Dystrophy who've been in a previous Givinostat study. They must have specific muscle fat levels, be able to consent, and use contraception if needed. Excluded are those with hypersensitivity to the drug's components, certain intolerances or diseases, heart failure, liver issues, psychiatric conditions affecting compliance, recent non-steroid muscle treatments, other neurological disorders or abnormal blood tests.

Inclusion Criteria

Must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit or must have been screened in study DSC/14/2357/48 and met all the inclusion criteria and none of the exclusion criteria

Never been randomized because the enrollment in the off-target group was completed

I am 6 years old or older.

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Exclusion Criteria

I am not using any experimental drugs except Givinostat.

I do not have any other serious illnesses that could interfere with the study.

Baseline QTcF >450 msec or history of additional risk factors for torsades de pointes

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Treatment Details

Interventions

Givinostat (Histone Deacetylase Inhibitor)

Trial OverviewThe trial is testing the long-term safety and effectiveness of Givinostat in patients with Duchenne Muscular Dystrophy. It's an open-label study meaning everyone knows they're getting Givinostat and it focuses on those who've previously taken part in related studies.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: givinostatExperimental Treatment1 Intervention

Givinostat oral suspension (10 mg/mL) twice daily in a fed state