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

NCT03238235 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 51

Last updated 2024-11-18

Study results available
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Summary

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.

Conditions

  • Becker Muscular Dystrophy

Interventions

DRUG

Givinostat

suspension of Givinostat (10 mg/mL)

DRUG

Placebo

suspension manufactured to mimic Givinostat

Sponsors & Collaborators

  • Italfarmaco

    lead INDUSTRY

Principal Investigators

  • Giacomo Comi, MD · Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-01-09
Primary Completion
2021-03-19
Completion
2021-03-19

Countries

  • Italy
  • Netherlands

Study Locations

More Related Trials

Entities

Drugs

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 NCT03238235 on ClinicalTrials.gov