At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Givinostat Hydrochloride (drug), Givinostat Hydrochloride - Cohort 2 (drug)
- How long the study runs
- Study runs about 59 months (dates as stated)
- About the drug or intervention
- Givinostat Hydrochloride — drug: Cohort 1 - from 4 to 6 years old · Givinostat Hydrochloride - Cohort 2 — drug: Cohort 2 - from 2 to 4 years old
- Patient visit burden
- Not specified by the sponsor
In plain English
This study looks at how the body handles a medicine called givinostat and how safe it is in boys with Duchenne muscular dystrophy (DMD) aged 2 to under 6 years. DMD is a genetic condition that causes muscle weakness. The study is paid for by a company called Italfarmaco.
Who can take part
- Boys aged 2 to under 6 years at screening, with a genetic diagnosis of Duchenne muscular dystrophy
- A parent or legal guardian must give written permission, and the child gives written agreement if that applies under local rules
- If taking steroid tablets, the dose must have been steady for at least 3 months before starting the study drug (changes for weight growth are allowed)
- If not taking steroids, the child must not start them during the first 48 weeks of the study
- For the extension phase: must have finished the 48-week main phase and the end-of-treatment visit, with permission given again, and be on a steady steroid dose (steroids may be started if not taken before, based on the doctor's judgement)
Who may not be able to
- Took another experimental drug within 3 months before starting the study drug
- Took any dystrophin restoration medicine (for example ataluren or exon skipping) within 6 months before starting
- Had any gene therapy (for example AAV micro-dystrophin) within 12 months before starting
- Took any non-steroid medicine that could affect muscle strength or function within 3 months before starting (for example growth hormone). Vitamin D, calcium and other supplements are allowed
- Had surgery in the last 3 months that could affect muscle strength or function, or surgery is planned during the study
- Has another significant illness or uncontrolled condition that, in the doctor's view, could affect safety or the ability to complete the study
- Blood counts below the normal range (platelets, white blood cells, or haemoglobin) at screening
- Current or past liver disease or impairment, including high bilirubin, unless due to Gilbert's syndrome
- Poor kidney function based on a blood test (serum Cystatin C more than twice the upper limit of normal)
- Fasting triglycerides (a blood fat) above 300 mg/dL (3.42 mmol/L) at screening
- Positive test for hepatitis B, hepatitis C, or HIV at screening
- A heart rhythm measure (QTcF) over 450 milliseconds on heart tracing, or extra risk factors for a serious heart rhythm problem (heart failure, low potassium, or family history of long QT syndrome)
- A mental health or social situation that, in the doctor's view, means the child could not follow the muscle tests or study procedures
- Allergic to any part of the study drug
- Cannot tolerate sorbitol, has sorbitol malabsorption, or has the inherited form of fructose intolerance
- Body weight under 10 kg at screening
- For the extension phase: low blood counts, liver problems, poor kidney function, or high fasting triglycerides at the end of treatment, or another significant illness or mental health or social situation affecting ability to take part
What taking part involves
- • Taking the study drug givinostat — how it is taken and the dose is not stated, ask the trial team
- • A main study phase lasting 48 weeks, followed by an extension phase
- • Muscle function tests and study procedures — details of what happens at visits are not stated, ask the trial team
Time commitment: The main phase lasts 48 weeks with an end-of-treatment visit, followed by an optional extension phase; full details of visits and tests are not stated — ask the trial team.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 2 Years to 6 Years
- Who
- Male
- Number of participants
- 18
- Started
- 2025-01-02
- Last checked
- 2025-07
Plain English Summary
What is this study?
- • Testing a new treatment for duchenne muscular dystrophy
- • Phase2 - 18 participants
- • 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
Who can take part?
- • Ages 2 Years to 6 Years
- • Diagnosed with duchenne muscular dystrophy
- • Male only
Where?
- • Leeds - Leeds Teaching Hospital NHS Trust
- • London - Great Ormond Street Hospital - GOSH
- • Newcastle upon Tyne - Newcastle upon Tyne Hospitals NHS Foundation Trust - Newcastle University
- • Oxford - Oxford University Hospitals NHS Foundation Trust
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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)
More detail
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).
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 2 Years - 6 Years
- Who can join: Male only
Treatment history
Treatments you must have had:
- ✓ participated in the Core Phase study (48 weeks) and have attended the End of Treatment Visit
What the study is looking for
- ✓Inclusion Criteria - Core Phase:
- ✓Male children aged ≥2 to \<6 years at screening (subjects ≥6 years of age at screening will not be enrolled into the...
- ✓Written consent provided by parent/legal guardian and subject written assent, if applicable (according to local...
- ✓A genetic diagnosis of DMD
- ✓Corticosteroid treatment considerations:
See the full criteria
Where Is This Study? (4 UK sites)
Leeds Teaching Hospital NHS Trust
Leeds, United Kingdom
Great Ormond Street Hospital - GOSH
London, United Kingdom
Newcastle upon Tyne Hospitals NHS Foundation Trust - Newcastle University
Newcastle upon Tyne NE1 eBZ, United Kingdom
Oxford University Hospitals NHS Foundation Trust
Oxford OX3 9DU, United Kingdom
How to Get in Touch
Italfarmaco Patient Advocacy
Sponsor contactCONTACT
Italfarmaco Patient Advocacy
Sponsor contactCONTACT
