Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy

Sponsor
Italfarmaco
Study ID
NCT03238235
Phase
PHASE2
Status
Completed

Conditions

  • Becker Muscular Dystrophy

Eligibility Criteria

Sex
MALE
Age
18 Years - 65 Years
Healthy Volunteers
Not accepted

Interventions

  • Givinostat — DRUG
    suspension of Givinostat (10 mg/mL)
  • Placebo — DRUG
    suspension manufactured to mimic Givinostat

Study Details

Objectives: Primary objective: to establish the histological effects of Givinostat versus placebo administered over 12 months. Secondary Objectives: * To establish the macroscopic muscle effects of Givinostat versus placebo administered over 12 months assessed by Magnetic Resonance Imaging (MRI)/Magnetic Resonance Spectroscopy (MRS). * To determine the other histological effects of Givinostat versus placebo administered over 12 months. * To establish the efficacy of Givinostat versus placebo administered chronically over 12 months in slowing disease progression. * To assess the safety and tolerability of Givinostat versus placebo administered chronically. * To evaluate the pharmacokinetic (PK) profile of Givinostat administered chronically in the target population. * To evaluate the impact of Givinostat versus placebo administered chronically on quality of life and activities of daily living.

Key Dates

Start date
Jan 9, 2018
Status verified
Mar 2024
Primary completion
Mar 19, 2021
Completion
Mar 19, 2021

Study Design

Enrollment
51 participants (actual)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Givinostat
    Givinostat oral suspension (10 mg/mL) twice daily in a fed state
  • Placebo Comparator: Placebo
    Placebo oral suspension (10 mg/mL) twice daily in a fed state

Primary Outcome Measure

Mean Change From Baseline to Visit 11 in Total Fibrosis (%) on Log Scale, Comparing the Histology of Muscle Biopsies [ Time Frame: after 12 months of treatment (at Visit 11) ]

Related Studies