Duchenne Muscular DystrophyPhase 2NCT06769633
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
A Phase 2 study testing Givinostat Hydrochloride and Givinostat Hydrochloride - Cohort 2 in people with Duchenne Muscular Dystrophy, run by Italfarmaco at 9 sites in 4 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 2 to 6 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Givinostat Hydrochloride and Givinostat Hydrochloride - Cohort 2
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 9 sitesin 4 countries
- Study dates
- 2 Jan 2025to Dec 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 6 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Male children aged ≥2 to <6 years at screening (subjects ≥6 years of age at screening will not be enrolled into the study)
- Written consent provided by parent/legal guardian and subject written assent, if applicable (according to local regulation)
- A genetic diagnosis of DMD
- Corticosteroid treatment considerations:
- For subject receiving a stable dose or oral systemic corticosteroids:
- No significant change in dose or dosing regimen (except for adjustments due to body weight change) for a minimum of 3 months immediately prior to the start of the study drug or
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- For subjects without current corticosteroid treatment:
- Must not start corticosteroids in the Core Phase of the study (ie, first 48 weeks).
- Must have participated in the Core Phase study (48 weeks) and have attended the End of Treatment Visit
- Give informed consent and /or assent in writing signed by the parent/legal guardian and/or subject (according to local regulation)
- In stable oral systemic corticosteroids treatment with no significant change in dose or dosing regimen (except for adjustments due to body weight change). For subjects without corticosteroids during the Core Phase, the treatment can be started based on the Investigator's clinical medical judgement.
Who cannot join
- Exposure to another investigational drug within 3 months prior to the start of the study drug
- Exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study drug
- Received any gene therapy (eg, AAV Micro-dystrophin delivery) within 12 months prior to start of study drug
- Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to the start of the study drug (eg, growth hormone). Note: Vitamin D, calcium, and any other supplements will be allowed.
- Have had surgery that might have an effect on muscle strength or function within 3 months prior to start of the study drug or planned surgery at any time during the study
- The presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect subject's safety, making it unlikely to complete the study or to be compliant with study-specific requirements that could impair the assessment of study results
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- Diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD, based on Investigator clinical medical judgement
- Platelet count, white blood cells, and/or haemoglobin counts < lower limit of normal (LLN) at screening (Note: for abnormal screening laboratory test results [<LLN], the platelet count, white blood cell, and haemoglobin will be repeated once; if the repeat test result is still <LLN, the subject will be excluded)
- Current or history of liver disease or impairment, including but not limited to a baseline elevated total bilirubin (ie, >1.5 × upper limit of normal [ULN]), unless secondary to Gilbert disease or pattern consistent with Gilbert disease
- Inadequate renal function, as defined by serum Cystatin C result >2 × ULN (Note: if the value is >2 × ULN, the serum Cystatin C will be repeated once; if the repeated test result is still >2 × ULN, the subject will be excluded)
- Fasting triglycerides >300 mg/dL (3.42 mmol/L) at screening (Note: if the value is >300 mg/dL, the triglycerides will be repeated once; if the repeated test result is still >300 mg/dL in fasting, the subject should be excluded)
- Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening
- Baseline corrected QT interval using Fridericia's formula (QTcF) >450 msec (as the mean of 3 consecutive readings taken 5 minutes apart) or history of additional risk factors for torsades de pointes (ie, heart failure, hypokalaemia, or family history of long QT syndrome)
- Psychiatric illness or social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures, based on the Investigator's clinical medical judgement
- Hypersensitivity to any component of study drug
- Sorbitol intolerance or malabsorption or have the hereditary form of fructose intolerance.
- Body weight <10 kg at screening.
- Platelet count, white blood cells, and/or haemoglobin <LLN at EOT/V12 (Note: for abnormal laboratory test results [<LLN], the platelet count, white blood cell, and haemoglobin will be repeated once; if the repeat test result is still <LLN, the subject will be excluded)
- Current liver disease or impairment, including but not limited to an elevated total bilirubin (ie, >1.5 × ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert disease
- Inadequate renal function, as defined by serum Cystatin C result >2 × ULN (Note: if the value is >2 × ULN, the serum Cystatin C will be repeated once; if the repeated test result is still >2 × ULN, the subject will be excluded)
- Fasting triglycerides >300 mg/dL (3.42 mmol/L; Note: if the value is >300 mg/dL, the triglycerides will be repeated once; if the repeated test result is still >300 mg/dL in fasting condition, the subject should be excluded)
- Have presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results
- Evidence of psychiatric illness or social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures, based on the Investigator's clinical medical judgement.
Trial locations and contacts
9 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
9 sites in 4 countries
United Kingdom4 sites, 4 recruiting
- Not yet recruiting
Leeds Teaching Hospital NHS Trust
Leeds, England
Anne-Marie Childs, MD, anne-marie.childs@nhs.net
- Not yet recruiting
Great Ormond Street Hospital - GOSH
London, England
Mariacristina Scoto, MD, Mariacristina Scoto <Mariacristina.Scoto@gosh.nhs.uk>
- Recruiting
Newcastle upon Tyne Hospitals NHS Foundation Trust - Newcastle University
Newcastle upon Tyne, England, NE1 eBZ
Michela Guglieri, MD, Michela Guglieri <michela.guglieri@newcastle.ac.uk>
- Recruiting
Oxford University Hospitals NHS Foundation Trust
Oxford, England, OX3 9DU
Laurent Servais, MD, Laurent Servais <laurent.servais@paediatrics.ox.ac.uk>
Italy3 sites, 3 recruiting
- Recruiting
Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca' Granda - NeuroMuscolar Omnicenter
Milan, 20162
Valeria Sansone, MD, valeria.sansone@centrocliniconemo.it
- Recruiting
Ospedale Pediatrico Bambino Gesù
Roma, 00165
Adele D'Amico, MD, +39 06 68592105, adele2.damico@opbg.net
- Recruiting
Policlinico Universitario Agostino Gemelli - Università Cattolica del Sacro Cuore
Roma, 00165
Eugenio Mercuri, MD, +39 06 30158576, eugeniomaria.mercuri@policlinicogemelli.it
Belgium1 site, 1 recruiting
- Recruiting
Queen Fabiola Children's University Hospital HUDERF
Brussels, 1020
Nicolas Deconinck, MD, +32 2 4772945, nicolas.deconink@hubruxelles.be
Netherlands1 site, 1 recruiting
- Recruiting
Leids Universitair Medisch Centrum (LUMC)
Leiden, 2300 RC
Erik Niks, MD, duchenne@lumc.nl
About this study
Official title: A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old
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)
Read the detailed description
This is an open-label, multicentre, multicountry, 2 cohorts study to evaluate the PK profile and safety of givinostat in subjects with DMD aged ≥4 to <6 years for Cohort 1 and aged ≥2 to <4 years for Cohort 2. The starting dose for Cohort 2 will be confirmed/adjusted with results of the interim analysis of Cohort 1. The study will consist of 2 phases, a Core Phase and an Extension Phase. Two final analyses will be conducted, the first at the end of the Core Phase and the second at the end of the Extension Phase (core and extension data will be combined).
The study will enrol approximately 18 subjects (approximately 9 subjects [aged ≥4 to <6 years] in Cohort 1 and approximately 9 subjects [aged ≥2 to <4 years] in Cohort 2).
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Givinostat Hydrochloride, Givinostat Hydrochloride - Cohort 2
- Keywords
- Duchenne Muscular Dystrophy, Givinostat
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Givinostat HydrochlorideDrug
Also called Cohort 1
Cohort 1 - from 4 to 6 years old
Givinostat Hydrochloride - Cohort 2Drug
Also called Cohort 2
Cohort 2 - from 2 to 4 years old
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 - from 4 to 6 years old | Experimental | Drug: Givinostat Givinostat has to be administered twice daily in a fed state according to a flexible dose regimen based on patient weight. Starting dose could be reduced based on predefined safety rules.
Other Names:
- ITF2357 Receives: Drug: Givinostat Hydrochloride |
| Cohort 2 - from 2 to 4 years old | Experimental | Drug: Givinostat Givinostat has to be administered twice daily in a fed state according to a flexible dose regimen based on patient weight. Starting dose will be confirmed/adjusted with results of the interim analysis of cohort 1.
Other Names:
- ITF2357 Receives: Drug: Givinostat Hydrochloride - Cohort 2 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Core Phase: Cohort 1 - Change from baseline of the pharmacokinetic (PK) parameter Area under the concentration-time curve from dosing (time 0) to time t at steady state (AUC0-T,ss).
Pharmacokinetic samples will be collected 1 pre-dose in the morning and 3 post-dose (0-2 h, 4-6 h, 6-8 h) after 1 week (Visit 3) and after 6 months (Visit 10). Sampling times will be randomly assigned to each subject to collect at least 1 pre-dose sample and 2 post-dose samples in each subject. At these timepoints, the subject will be asked to take the morning dose at the site. Samples collected after 1 week of treatment will be used for the exploratory PK analysis.
Measured: Baseline up to week 48
Core Phase: Cohort 1 - Change from baseline of the pharmacokinetic (PK) parameter maximum plasma concentration at steady state (Cmax,ss).
Pharmacokinetic samples will be collected 1 pre-dose in the morning and 3 post-dose (0-2 h, 4-6 h, 6-8 h) after 1 week (Visit 3) and after 6 months (Visit 10). Sampling times will be randomly assigned to each subject to collect at least 1 pre-dose sample and 2 post-dose samples in each subject. At these timepoints, the subject will be asked to take the morning dose at the site. Samples collected after 1 week of treatment will be used for the exploratory PK analysis.
Measured: Baseline up to week 48
Core Phase: Cohort 1 - Change from baseline of the pharmacokinetic (PK) parameter elimination half-life (t1/2) assessed after at least 7 days of dosing.
Pharmacokinetic samples will be collected 1 pre-dose in the morning and 3 post-dose (0-2 h, 4-6 h, 6-8 h) after 1 week (Visit 3) and after 6 months (Visit 10). Sampling times will be randomly assigned to each subject to collect at least 1 pre-dose sample and 2 post-dose samples in each subject. At these timepoints, the subject will be asked to take the morning dose at the site. Samples collected after 1 week of treatment will be used for the exploratory PK analysis.
Measured: Baseline up to week 48
Core Phase: Cohort 2 - Change from baseline of the pharmacokinetic (PK) parameter Area under the concentration-time curve from dosing (time 0) to time t at steady state (AUC0-T,ss).
Pharmacokinetic samples will be collected 1 pre-dose in the morning and 3 post-dose (0-2 h, 4-6 h, 6-8 h) after 1 week (Visit 3) and after 6 months (Visit 10). Sampling times will be randomly assigned to each subject to collect at least 1 pre-dose sample and 2 post-dose samples in each subject. At these timepoints, the subject will be asked to take the morning dose at the site. Samples collected after 1 week of treatment will be used for the exploratory PK analysis.
Measured: Baseline up to week 48
Core Phase: Cohort 2 - Change from baseline of the pharmacokinetic (PK) parameter maximum plasma concentration at steady state (Cmax,ss).
Pharmacokinetic samples will be collected 1 pre-dose in the morning and 3 post-dose (0-2 h, 4-6 h, 6-8 h) after 1 week (Visit 3) and after 6 months (Visit 10). Sampling times will be randomly assigned to each subject to collect at least 1 pre-dose sample and 2 post-dose samples in each subject. At these timepoints, the subject will be asked to take the morning dose at the site. Samples collected after 1 week of treatment will be used for the exploratory PK analysis.
Measured: Baseline up to week 48
Core Phase: Cohort 2 - Change from baseline of the pharmacokinetic (PK) parameter elimination half-life (t1/2) assessed after at least 7 days of dosing
Pharmacokinetic samples will be collected 1 pre-dose in the morning and 3 post-dose (0-2 h, 4-6 h, 6-8 h) after 1 week (Visit 3) and after 6 months (Visit 10). Sampling times will be randomly assigned to each subject to collect at least 1 pre-dose sample and 2 post-dose samples in each subject. At these timepoints, the subject will be asked to take the morning dose at the site. Samples collected after 1 week of treatment will be used for the exploratory PK analysis.
Measured: Baseline up to week 48
Extension Phase: Type, incidence, and severity of treatment-emergent adverse events
Measured: postbaseline up to Week 144
Extension Phase: Proportion of patients experiencing treatment-emergent adverse events
Measured: postbaseline up to Week 144
Secondary outcomes (5)
Core Phase: Type, incidence, and severity of treatment-emergent adverse events
Measured: Baseline up to week 48
Core Phase: Proportion of patients experiencing treatment-emergent adverse events
Measured: Baseline up to week 48
Core Phase: Change from baseline as measured by North Star Ambulatory Assessment (NSAA) in cohort 1 after 48 weeks of treatment of givinostat.
Measured: Baseline up to week 48
Core Phase: Change from baseline as measured by Bayley III Gross Motor in cohort 2 after 48 weeks of treatment of givinostat.
Measured: Baseline up to week 48
Core Phase: Change from baseline in quality of life (as measured by health-related quality of life, HRQOL) at week 48 of treatment of givinostat
Measured: Baseline up to week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2025
- Study start2 Jan 2025
- Primary completionDec 2029 (estimated)
- Study completionDec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Italfarmaco
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Fortrea
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06769633
- Sponsor's protocol ID
- DSC/14/2357/52
- Status last verified
- Jul 2025
- First submitted
- 11 Dec 2024
- Last updated
- 10 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old
- ClinicalTrials.gov ID
- NCT06769633
- Acronym / sponsor ID
- DSC/14/2357/52
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, Givinostat
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- ItalfarmacoIndustry (pharma, biotech, devices)
- Collaborators
- Fortrea
- Study start
- 2 Jan 2025 (actual)
- Primary completion
- Dec 2029 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 10 Jan 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Detailed description
This is an open-label, multicentre, multicountry, 2 cohorts study to evaluate the PK profile and safety of givinostat in subjects with DMD aged ≥4 to <6 years for Cohort 1 and aged ≥2 to <4 years for Cohort 2. The starting dose for Cohort 2 will be confirmed/adjusted with results of the interim analysis of Cohort 1. The study will consist of 2 phases, a Core Phase and an Extension Phase. Two final analyses will be conducted, the first at the end of the Core Phase and the second at the end of the Extension Phase (core and extension data will be combined).
The study will enrol approximately 18 subjects (approximately 9 subjects [aged ≥4 to <6 years] in Cohort 1 and approximately 9 subjects [aged ≥2 to <4 years] in Cohort 2).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 - from 4 to 6 years old | Experimental | Drug: Givinostat HydrochlorideDrug: Givinostat Givinostat has to be administered twice daily in a fed state according to a flexible dose regimen based on patient weight. Starting dose could be reduced based on predefined safety rules. Other Names: - ITF2357 |
| Cohort 2 - from 2 to 4 years old | Experimental | Drug: Givinostat Hydrochloride - Cohort 2Drug: Givinostat Givinostat has to be administered twice daily in a fed state according to a flexible dose regimen based on patient weight. Starting dose will be confirmed/adjusted with results of the interim analysis of cohort 1. Other Names: - ITF2357 |
Who may qualify
- Ages
- 2 to 6 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- Male children aged ≥2 to <6 years at screening (subjects ≥6 years of age at screening will not be enrolled into the study)
- Written consent provided by parent/legal guardian and subject written assent, if applicable (according to local regulation)
- A genetic diagnosis of DMD
- Corticosteroid treatment considerations:
- No significant change in dose or dosing regimen (except for adjustments due to body weight change) for a minimum of 3 months immediately prior to the start of the study drug or
Key exclusion criteria (23 listed)
- Exposure to another investigational drug within 3 months prior to the start of the study drug
- Exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study drug
- Received any gene therapy (eg, AAV Micro-dystrophin delivery) within 12 months prior to start of study drug
- Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to the start of the study drug (eg, growth hormone). Note: Vitamin D, calcium, and any other supplements will be allowed.
- Have had surgery that might have an effect on muscle strength or function within 3 months prior to start of the study drug or planned surgery at any time during the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Givinostat HydrochlorideAlso: Cohort 1Cohort 1 - from 4 to 6 years old | Drug | Cohort 1 - from 4 to 6 years old |
| Givinostat Hydrochloride - Cohort 2Also: Cohort 2Cohort 2 - from 2 to 4 years old | Drug | Cohort 2 - from 2 to 4 years old |
Primary outcome
Core Phase: Cohort 1 - Change from baseline of the pharmacokinetic (PK) parameter Area under the concentration-time curve from dosing (time 0) to time t at steady state (AUC0-T,ss).
Time frame: Baseline up to week 48
Pharmacokinetic samples will be collected 1 pre-dose in the morning and 3 post-dose (0-2 h, 4-6 h, 6-8 h) after 1 week (Visit 3) and after 6 months (Visit 10). Sampling times will be randomly assigned to each subject to collect at least 1 pre-dose sample and 2 post-dose samples in each subject. At these timepoints, the subject will be asked to take the morning dose at the site. Samples collected after 1 week of treatment will be used for the exploratory PK analysis.
Core Phase: Cohort 1 - Change from baseline of the pharmacokinetic (PK) parameter maximum plasma concentration at steady state (Cmax,ss).
Time frame: Baseline up to week 48
Pharmacokinetic samples will be collected 1 pre-dose in the morning and 3 post-dose (0-2 h, 4-6 h, 6-8 h) after 1 week (Visit 3) and after 6 months (Visit 10). Sampling times will be randomly assigned to each subject to collect at least 1 pre-dose sample and 2 post-dose samples in each subject. At these timepoints, the subject will be asked to take the morning dose at the site. Samples collected after 1 week of treatment will be used for the exploratory PK analysis.
Core Phase: Cohort 1 - Change from baseline of the pharmacokinetic (PK) parameter elimination half-life (t1/2) assessed after at least 7 days of dosing.
Time frame: Baseline up to week 48
Pharmacokinetic samples will be collected 1 pre-dose in the morning and 3 post-dose (0-2 h, 4-6 h, 6-8 h) after 1 week (Visit 3) and after 6 months (Visit 10). Sampling times will be randomly assigned to each subject to collect at least 1 pre-dose sample and 2 post-dose samples in each subject. At these timepoints, the subject will be asked to take the morning dose at the site. Samples collected after 1 week of treatment will be used for the exploratory PK analysis.
Core Phase: Cohort 2 - Change from baseline of the pharmacokinetic (PK) parameter Area under the concentration-time curve from dosing (time 0) to time t at steady state (AUC0-T,ss).
Time frame: Baseline up to week 48
Pharmacokinetic samples will be collected 1 pre-dose in the morning and 3 post-dose (0-2 h, 4-6 h, 6-8 h) after 1 week (Visit 3) and after 6 months (Visit 10). Sampling times will be randomly assigned to each subject to collect at least 1 pre-dose sample and 2 post-dose samples in each subject. At these timepoints, the subject will be asked to take the morning dose at the site. Samples collected after 1 week of treatment will be used for the exploratory PK analysis.
Core Phase: Cohort 2 - Change from baseline of the pharmacokinetic (PK) parameter maximum plasma concentration at steady state (Cmax,ss).
Time frame: Baseline up to week 48
Pharmacokinetic samples will be collected 1 pre-dose in the morning and 3 post-dose (0-2 h, 4-6 h, 6-8 h) after 1 week (Visit 3) and after 6 months (Visit 10). Sampling times will be randomly assigned to each subject to collect at least 1 pre-dose sample and 2 post-dose samples in each subject. At these timepoints, the subject will be asked to take the morning dose at the site. Samples collected after 1 week of treatment will be used for the exploratory PK analysis.
Core Phase: Cohort 2 - Change from baseline of the pharmacokinetic (PK) parameter elimination half-life (t1/2) assessed after at least 7 days of dosing
Time frame: Baseline up to week 48
Pharmacokinetic samples will be collected 1 pre-dose in the morning and 3 post-dose (0-2 h, 4-6 h, 6-8 h) after 1 week (Visit 3) and after 6 months (Visit 10). Sampling times will be randomly assigned to each subject to collect at least 1 pre-dose sample and 2 post-dose samples in each subject. At these timepoints, the subject will be asked to take the morning dose at the site. Samples collected after 1 week of treatment will be used for the exploratory PK analysis.
Extension Phase: Type, incidence, and severity of treatment-emergent adverse events
Time frame: postbaseline up to Week 144
Extension Phase: Proportion of patients experiencing treatment-emergent adverse events
Time frame: postbaseline up to Week 144
5 secondary outcomes
Core Phase: Type, incidence, and severity of treatment-emergent adverse events
Time frame: Baseline up to week 48
Core Phase: Proportion of patients experiencing treatment-emergent adverse events
Time frame: Baseline up to week 48
Core Phase: Change from baseline as measured by North Star Ambulatory Assessment (NSAA) in cohort 1 after 48 weeks of treatment of givinostat.
Time frame: Baseline up to week 48
Core Phase: Change from baseline as measured by Bayley III Gross Motor in cohort 2 after 48 weeks of treatment of givinostat.
Time frame: Baseline up to week 48
Core Phase: Change from baseline in quality of life (as measured by health-related quality of life, HRQOL) at week 48 of treatment of givinostat
Time frame: Baseline up to week 48
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
9 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Givinostat Hydrochloride and Givinostat Hydrochloride - Cohort 2 in people with Duchenne Muscular Dystrophy, run by Italfarmaco at 9 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United Kingdom, Italy, Belgium, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Italfarmaco Patient Advocacy (+39 02 64431, patientadvocacy@italfarmacogroup.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Core Phase: Cohort 1 - Change from baseline of the pharmacokinetic (PK) parameter Area under the concentration-time curve from dosing (time 0) to time t at steady state (AUC0-T,ss). (time frame: Baseline up to week 48). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jul 2025.