A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy

A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy - DSC

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2012-002566-12-IT

Enrollment

Registered

2012-10-17

Start date

2013-02-04

Completion date

Unknown

Last updated

2018-02-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy MedDRA version: 14.1 Level: SOC Classification code 10028395 Term: Musculoskeletal and connective tissue disorders System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders

Interventions

Product Name: Givinostat Product Code: ITF2357 Pharmaceutical Form: Oral suspension INN or Proposed INN: Givinostat CAS Number: 732302-99-7 Current Sponsor code: ITF2357 Other descriptive name: givino

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: 1. Male children aged 7 to =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. Initiation of systemic corticosteroid therapy within 6 months prior to the start of study drug or change in systemic corticosteroid therapy (e.g., initiation, change in type of drug, dose modification not related to body weight change, schedule modification, interruption, discontinuation, or re initiation) within 6 months prior to the start of study drug. 2. Use of any pharmacologic treatment, other than corticosteroids, that might have an effect on muscle strength since the time of the historical 6MWT and in any case within 3 months prior to the start of study treatment (e.g., growth hormone). Vitamin D, calcium, and integrators will be allowed. 3. Surgery that might have an effect on muscle strength or function within 3 months before study entry or planned surgery at any time during the study.

Design outcomes

Primary

Measure	Time frame
Main Objective: To establish the histologic effects of Givinostat administered chronically at the selected daily dose;Secondary Objective: • To establish the effects of Givinostat administered chronically at the selected daily dose on functional parameters, such as the 6-Minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), and performance of upper limb (PUL) • To establish the safety and tolerability of Givinostat administered chronically at the selected daily dose in children with Duchenne muscular dystrophy (DMD) • To explore the effects of Givinostat administered chronically at the selected daily dose on parameters such as magnetic resonance imaging (MRI), biomarkers, and cytokines;Primary end point(s): The primary endpoint of this study is change in the value of MFA% comparing the histology biopsies before and after 12 months of treatment with Givinostat.;Timepoint(s) of evaluation of this end point: 12 months	—

Measure

Time frame

Main Objective: To establish the histologic effects of Givinostat administered chronically at the selected daily dose;Secondary Objective: • To establish the effects of Givinostat administered chronically at the selected daily dose on functional parameters, such as the 6-Minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), and performance of upper limb (PUL) • To establish the safety and tolerability of Givinostat administered chronically at the selected daily dose in children with Duchenne muscular dystrophy (DMD) • To explore the effects of Givinostat administered chronically at the selected daily dose on parameters such as magnetic resonance imaging (MRI), biomarkers, and cytokines;Primary end point(s): The primary endpoint of this study is change in the value of MFA% comparing the histology biopsies before and after 12 months of treatment with Givinostat.;Timepoint(s) of evaluation of this end point: 12 months

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Secondary

Measure	Time frame
Secondary end point(s): • Change in additional histological endpoints (i.e., cross-sectional area, inflammation, necrosis, fibrosis, and muscle regeneration) after 12 months of treatment with Givinostat at the selected daily dose • Change in muscular function after 12 months of treatment with Givinostat at the selected daily dose based on the 6MWT • Change in muscular function after 12 months of treatment with Givinostat at the selected daily dose based on the NSAA • Change in muscular function after 12 months of treatment with Givinostat at the selected daily dose based the PUL;Timepoint(s) of evaluation of this end point: 12 months	—

Measure

Time frame

Secondary end point(s): • Change in additional histological endpoints (i.e., cross-sectional area, inflammation, necrosis, fibrosis, and muscle regeneration) after 12 months of treatment with Givinostat at the selected daily dose • Change in muscular function after 12 months of treatment with Givinostat at the selected daily dose based on the 6MWT • Change in muscular function after 12 months of treatment with Givinostat at the selected daily dose based on the NSAA • Change in muscular function after 12 months of treatment with Givinostat at the selected daily dose based the PUL;Timepoint(s) of evaluation of this end point: 12 months

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Countries

Italy

Contacts

Public ContactClinical R&D Dept.

Italfarmaco

b.gatti@italfarmaco.com02 6443 2512

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026