Actively Recruiting
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
Led by Italfarmaco · Updated on 2025-07-10
18
Participants Needed
9
Research Sites
256 weeks
Total Duration
On this page
Sponsors
I
Italfarmaco
Lead Sponsor
F
Fortrea
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in younger DMD Patients. * Planned screening duration: approximately 4 weeks * Planned Core Treatment duration: approximately 48 weeks * Planned Extension Treatment duration: approximately 96 weeks * Planned Follow Up duration: approximately 4 weeks (± 7 days) * Total duration of study participation: up to 151 weeks (ie, 37-38 months)
CONDITIONS
Official Title
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male children aged 2 to less than 6 years at screening
- Written consent from parent/legal guardian and assent from participant if applicable
- Genetic diagnosis of Duchenne Muscular Dystrophy
- Stable corticosteroid treatment for at least 3 months prior to study or no corticosteroid treatment and not starting corticosteroids during Core Phase
- For Extension Phase: participation in Core Phase and attendance at End of Treatment Visit
- For Extension Phase: informed consent/assent for extension participation
- For Extension Phase: stable corticosteroid treatment or corticosteroid treatment started based on Investigator's judgment
You will not qualify if you...
- Use of another investigational drug within 3 months prior to study
- Use of dystrophin restoration products within 6 months prior to study
- Receipt of gene therapy within 12 months prior to study
- Use of pharmacologic treatments affecting muscle strength (except corticosteroids) within 3 months prior to study
- Surgery affecting muscle strength within 3 months prior to study or planned during study
- Presence of other significant diseases affecting safety or study compliance
- Diagnosis of uncontrolled neurological diseases or relevant somatic disorders not related to DMD
- Low platelet count, white blood cells, or hemoglobin below normal at screening
- Current or history of liver disease or impairment except Gilbert disease
- Inadequate renal function as defined by serum Cystatin C above limits
- Fasting triglycerides above 300 mg/dL at screening
- Positive test for hepatitis B, hepatitis C, or HIV
- Corrected QT interval above 450 msec or risk factors for torsades de pointes
- Psychiatric illness or social situations preventing understanding or compliance
- Hypersensitivity to any study drug component
- Sorbitol intolerance, malabsorption, or hereditary fructose intolerance
- Body weight less than 10 kg at screening
- For Extension Phase: abnormal blood counts, liver disease, renal impairment, high triglycerides, significant disease affecting safety or compliance, or psychiatric illness preventing study compliance
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 9 locations
1
Queen Fabiola Children's University Hospital HUDERF
Brussels, Belgium, 1020
Actively Recruiting
2
Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca' Granda - NeuroMuscolar Omnicenter
Milan, Italy, 20162
Actively Recruiting
3
Ospedale Pediatrico Bambino Gesù
Roma, Italy, 00165
Actively Recruiting
4
Policlinico Universitario Agostino Gemelli - Università Cattolica del Sacro Cuore
Roma, Italy, 00165
Actively Recruiting
5
Leids Universitair Medisch Centrum (LUMC)
Leiden, Netherlands, 2300 RC
Actively Recruiting
6
Leeds Teaching Hospital NHS Trust
Leeds, England, United Kingdom
Not Yet Recruiting
7
Great Ormond Street Hospital - GOSH
London, England, United Kingdom
Not Yet Recruiting
8
Newcastle upon Tyne Hospitals NHS Foundation Trust - Newcastle University
Newcastle upon Tyne, England, United Kingdom, NE1 eBZ
Actively Recruiting
9
Oxford University Hospitals NHS Foundation Trust
Oxford, England, United Kingdom, OX3 9DU
Actively Recruiting
Research Team
I
Italfarmaco Patient Advocacy
CONTACT
I
Italfarmaco Patient Advocacy
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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