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 (drug)
- How long the study runs
- Study runs about 146 months (dates as stated)
- About the drug or intervention
- Givinostat — drug: suspension of givinostat (10 mg/mL)
- Patient visit burden
- Not specified by the sponsor
In plain English
This study looks at the long-term safety and how well tolerated Givinostat is in people with Duchenne Muscular Dystrophy (DMD). It is run by the sponsor Italfarmaco. It is mainly for people who took part in an earlier Givinostat study in DMD, or who were screened in a previous study but could not join.
Who can take part
- Must have taken part in a previous Givinostat study in Duchenne Muscular Dystrophy and attended the end of study visit, or have been screened in study DSC/14/2357/48, met all the entry rules, had a certain leg muscle fat measurement, but were not selected to join
- Aged 6 years or older
- Able to give agreement (assent) or consent in writing, signed by the person taking part and/or a parent or legal guardian, following local rules
- Willing to use suitable contraception, from the previous study until 3 months after the last dose of the study drug
Who may not be able to
- Taken any medicine (other than corticosteroids) that could affect muscle strength or function in the 3 months before joining, for example growth hormone (vitamin D, calcium and other supplements are allowed)
- Currently taking any other experimental (investigational) drug besides Givinostat
- Has another significant illness that, in the researcher's view, could affect safety, make it unlikely the person can finish the treatment or follow-up, or affect the study results
- Has other uncontrolled neurological conditions or significant uncontrolled health problems not related to Duchenne Muscular Dystrophy
- Has blood tests below the normal lower limit for platelets, white blood cells, or haemoglobin (if the first result is abnormal, the test is repeated once; if still low, the person cannot take part)
- Has fasting triglycerides (a type of fat in the blood) above 300 mg/dL (3.42 mmol/L) at screening (retested once if abnormal; still high means exclusion)
- Has poor kidney function, shown by a blood test (serum Cystatin C) more than twice the upper limit of normal (retested once if abnormal; still high means exclusion)
- Has heart failure classed as New York Heart Association Class III or IV
- Has a current liver disease or liver problem, including higher than normal total bilirubin (a liver blood test), unless caused by Gilbert's syndrome
- Has a heart tracing (QTcF) reading above 450 milliseconds, based on the average of 3 readings taken 5 minutes apart, or other risk factors for a serious heart rhythm problem
- Has a mental health condition or social situation that makes it hard to understand and carry out the muscle function tests or follow the study procedures
- Is allergic to any ingredient of the study medicine
- Has sorbitol intolerance or malabsorption, or the inherited condition fructose intolerance
What taking part involves
- • Taking the study drug Givinostat
- • The study team may check laboratory results and change the Givinostat dose if needed, following the study's safety and dose change rules
Time commitment: Not stated — ask the trial team about how many visits there are, how long the study lasts, and what taking part involves.
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
- 7 Years and over
- Who
- Male
- Number of participants
- 206
- Started
- 2017-10-24
- Last checked
- 2026-01
Plain English Summary
What is this study?
- • Testing a new treatment for duchenne muscular dystrophy
- • Phase2/Phase3 - 206 participants
- • This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies
Who can take part?
- • Ages 7 Years and over
- • Diagnosed with duchenne muscular dystrophy
- • Male only
Where?
- • Liverpool - Alder Hey Children's Hospital NHS Trust
- • Gobowen - The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust
- • London - UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD
- • Newcastle upon Tyne - The John Walton Muscular Dystrophy Research Centre
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.
More detail
GIVINOSTAT oral suspension (10 mg/mL) has to be administered orally as 2 oral doses daily while the subject is in a fed state. The starting dose of GIVINOSTAT in the present long term study will be the same that the subject was receiving at the end of the previous DMD GIVINOSTAT study. As weight affects GIVINOSTAT exposures, the dosage will be modified based on subject weight according the rules detailed in the study protocol section 11.2.2.1. In addition, in case a subject will have a consistent (e.g., at least 2 consecutive evaluations) platelets count ≤150 x 10\^9/L and not meet the stopping criteria for platelets, the Investigator will have to reduce the dose of 1/3 or 20% less of the current dose as described in the study protocol section 10.5.1.3. During the first month of treatment, platelets count assessment will be performed weekly, while during the second month it will be performed every 2 weeks, in order to strictly monitor this parameter for safety reasons, with the exclusion of subjects coming from study DSC/11/2357/43 for which the first visit will be 4 months after the Visit 1/baseline visit. Study drug should be permanently interrupted if any of the following occurs: * severe drug-related diarrhoea (i.e., increase of ≥7 stools per day); * any drug-related SAE; * QTcF \>500 msec; * platelets count ≤50 x 10\^9/L; * white blood cells ≤2.0 x 10\^9/L; * hemoglobin ≤8.0 g/dL; To avoid laboratory errors and anomalous values, test must be confirmed with a repeated test performed on the next working day. The treatment should be stopped until the retest result becomes available. If the repeated test is still under the stopping limit value, study drug must be permanently discontinued. If the repeated test is acceptable, the subject can resume treatment. The Investigator will follow up the patient until resolution or acceptable stabilization of the event occurs and document all the relevant information, as applicable. After the resolution/stabilization of the event, the subject will be withdrawn from the study and the EOS Visit (see Section 12.1.10) will be performed. Any decision relevant to the dose adjustment and/or modification of schedule of assessments can be discussed with the Medical Monitor, but the final decision remains with the Investigator only or its authorized designee.
How this trial compares with your answers
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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: 7 Years and over
- Who can join: Male only
Biomarkers mentioned
What the study is looking for
- ✓Must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study...
- ✓all the inclusion criteria and none of the exclusion criteria,
- ✓had a baseline vastus lateralis muscle fat fraction (VL MFF) assessed by MRS in the range ≤5% or \>30%, i.e....
- ✓never been randomly assigned because, the enrollment in the off target group was completed.
- ✓Aged ≥6 years old;
Who cannot take part
- ✗Use of any current investigational drug other than Givinostat;
- ✗Have a diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders...
- ✗Have heart failure (New York Heart Association Class III or IV)
- ✗Have a current liver disease or impairment, including but not limited to an elevated total liver blood test\* (i.e. \> 1.5...
- ✗Have a psychiatric illness/social situation rendering the potential subject unable to understand and comply with the...
See the full criteria
Where Is This Study? (4 UK sites)
Alder Hey Children's Hospital NHS Trust
Liverpool L12 2AP, United Kingdom
The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust
Gobowen SY10 7AG, United Kingdom
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD
London , WC1N 1EH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700The John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne NE1 3BZ, United Kingdom
How to Get in Touch
Reference Study ID Number: DSC/14/2357/51
Sponsor contactCONTACT
