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

Part of paid clinical trials in Davis, California.

Sponsor
Italfarmaco
Study ID
NCT02851797
Phase
PHASE3
Status
Completed

Conditions

Eligibility Criteria

Sex
MALE
Age
6 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • givinostat — DRUG
    The oral suspension of givinostat (10 mg/mL) was to be dosed in fed condition as described below: Givinostat or placebo starting dose * \> or =10 and \< 12.5 kg of weight: 13.3 mg bid = 1.3 ml oral suspension bid * \> or =12.5 and \< 20 kg: 16.7 mg bid =1.7 ml oral suspension bid * \> or = 20 and \< 25 kg: 20 mg bid = 2.0 ml oral suspension bid * \> or = 25 and \< 30 kg: 23.3 mg bid = 2.3 ml oral suspension bid * \> or = 30 and \< 40 kg: 26.7 mg bid = 2.7 ml oral suspension bid * \> or = 40 and \< 50 kg: 33.3 mg bid = 3.3 ml oral suspension bid * \> or = 50 and \< 60 kg: 36.7 mg bid = 3.7 ml oral suspension bid * \> or = 60 and \< 70 kg: 40 mg bid = 4 ml oral suspension bid * \> or = 70 kg: 46.7 mg bid = 4.7 ml oral suspension bid
  • placebo — DRUG
    The oral suspension of placebo, manufactured to mimic givinostat, was to be dosed in fed condition as described for givinostat.

Study Details

Primary Objective The primary objective of the study was to establish the effects of givinostat versus placebo administered chronically over 18 months to slow disease progression in ambulant DMD subjects. Secondary Objectives The secondary objectives of this study were: * To assess the safety and tolerability of givinostat versus placebo administered chronically in DMD subjects * To evaluate the PK profile of givinostat administered chronically in DMD subjects * To evaluate the impact on quality of life (QoL) and activities of daily living of givinostat versus placebo administered chronically.

Key Dates

Start date
Jun 6, 2017
Status verified
Jan 2023
Primary completion
Feb 22, 2022
Completion
Feb 22, 2022

Study Design

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

Arms

  • Active Comparator: givinostat
    Givinostat oral suspension (10 mg/mL) twice daily
  • Placebo Comparator: placebo
    Placebo oral suspension (10 mg/mL) twice daily

Primary Outcome Measure

Mean Change From Baseline in 4 Standard Stairs (4SC) Climb After 18 Months of Treatment [ Time Frame: Baseline and 18 months ]

Locations (13)

FacilityCityStateZIPSite coordinators
Neuromuscular Research Center UC Davis Department of Physical Medicine and RehabilitationDavisCalifornia95817-
Rady Children's Hospital center - UCSD Department of NeuroscienceSan DiegoCalifornia92123-
Children's Hospital ColoradoAuroraColorado80045-
Connecticut Children's Medical Center - Division NeurologyHartfordConnecticut06106-
Child Health Research Institute - Department of PediatricsGainesvilleFlorida32610-
Nemours Children's HospitalOrlandoFlorida32827-
MD Rare Disease Research, LLCAtlantaGeorgia30318-
University of Iowa Children's HospitalIowa CityIowa52242-
University of Minnesota - Department of NeurologyMinneapolisMinnesota55455-
Washington University School of Medicine in St Louis - Department of NeurologySt LouisMissouri63110-
Shriners Hospitals for ChildrenPortlandOregon97239-
The Children's Hospital of Philadelphia Colket Translational Research BuildingPhiladelphiaPennsylvania19104-
Virginia Commonwealth University Childrens Hospital of Richmond at Virginia Commonwealth UniversityRichmondVirginia23298-

Find similar trials in Davis, CA

By condition
Duchenne muscular dystrophy
By specialty
Pediatrics
By research site
Neuromuscular Research Center UC Davis Department of Physical Medicine and Rehabilitation· Davis, CARady Children's Hospital center - UCSD Department of Neuroscience· San Diego, CAChildren's Hospital Colorado· Aurora, COConnecticut Children's Medical Center - Division Neurology· Hartford, CTChild Health Research Institute - Department of Pediatrics· Gainesville, FLNemours Children's Hospital· Orlando, FL

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