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

NCT02851797 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 179

Last updated 2023-02-02

Study results available
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Summary

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.

Conditions

Interventions

DRUG

givinostat

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

DRUG

placebo

The oral suspension of placebo, manufactured to mimic givinostat, was to be dosed in fed condition as described for givinostat.

Sponsors & Collaborators

Principal Investigators

  • Eugenio Mercuri, MD, PhD · Istituto di Ricovero e Cura a Carattere Scientifico, IRCCS

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Max Age
17 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-06-06
Primary Completion
2022-02-22
Completion
2022-02-22
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Canada
  • France
  • Germany
  • Israel
  • Italy
  • Netherlands
  • Serbia
  • Spain
  • United Kingdom

Study Locations

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Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02851797 on ClinicalTrials.gov