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Looking for participantsPhase2/Phase3

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

Sponsor: Italfarmaco

NCT ID: NCT03373968

View on ClinicalTrials.gov ↗

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.

Duchenne Muscular Dystrophy

How this trial compares with your answers

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What we know so far

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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

met

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
Inclusion Criteria: 1. Must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit or must have been screened in study DSC/14/2357/48 and met: * 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. included in"off-target" group, * never been randomized because, the enrollment in the off target group was completed. 2. Aged ≥6 years old; 3. Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to localregulations); 4. Subjects must be willing to use adequate contraception: * Contraceptive methods must since the previous GIVINOSTAT study through 3 months after the last dose of study drug, and include the following: * True abstinence (absence of any sexual intercourse), when in line with the preferred and usual lifestyle of the subject. * Periodic abstinence (e.g. calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception. * Condom with spermicide and the female partner must use an acceptable method of contraception, such as an oral, * transdermal, injectable or implanted steroid-basedcontraceptive, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such asfor example cervical cap with spermicide jelly. Exclusion Criteria: 1. Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to be enrolled in this study (e.g., growth hormone); Vitamin D, calcium, and any other supplements will be allowed; 2. Use of any current investigational drug other than Givinostat; 3. 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; 4. Have a diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD; 5. Have platelets count, White Blood Cell and Hemoglobin at screening \< Lower Limit of Normal (LLN)\* (for abnormal screening laboratory test results (\<LLN), the platelets count, White Blood Cell and Hemoglobin will be repeated once; if the repeat test result is still \<LLN, then exclusionary); 6. Have Triglycerides \> 300 mg/dL (3.42 mmol/L) in fasting condition at screening visit\* (for abnormal screening laboratory test results (\>300 mg/dL), the triglycerides will be repeated once; if the repeat test result is still \>300 mg/dL, then exclusionary); 7. Have inadequate renal function, as defined by serum Cystatin C \>2 x the upper limit of normal (ULN) at screening visit\*. If the value is \>2 x ULN, the serum Cystatin C will be repeated once; if the repeated test result is still \>2 x ULN, the subject should be excluded); 8. Have heart failure (New York Heart Association Class III or IV) 9. Have a current liver disease or impairment, including but not limited to an elevated total bilirubin\* (i.e. \> 1.5 x ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert's; 10. Have a baseline QTcF \>450 msec, (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome); 11. Have a psychiatric illness/social situation rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures. 12. Have any hypersensitivity to the components of study medication; 13. Have a sorbitol intolerance or sorbitol malabsorption or have the hereditary form of fructose intolerance. * the Investigators to evaluate these exclusion criteria can use the laboratory results obtained within 5 months from V1, to allow the continuity of the treatment. It is worth noting, as soon as the site will receive the laboratory results done in screening/baseline (Visit 1) visit they will check the GIVINOSTAT dose and modify it as per protocol safety rules and/or dosage modifications rules.

Where Is This Study? (4 UK sites)

Alder Hey Children's Hospital NHS Trust

Liverpool L12 2AP, United Kingdom

ENROLLING_BY_INVITATION
Hospital R&D contact (matched)

Kelly Davies

research@alderhey.nhs.uk0151 2525570

The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust

Gobowen SY10 7AG, United Kingdom

ENROLLING_BY_INVITATION
Hospital R&D contact (matched)

Teresa Jones

rjah.researchoffice@nhs.net01691 404143

UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD

London , WC1N 1EH, United Kingdom

ENROLLING_BY_INVITATION
Hospital R&D contact (matched)

Main Email: Research.Governance@gosh.nhs.uk

Research.Governance@gosh.nhs.uk0207 905 2700

The John Walton Muscular Dystrophy Research Centre

Newcastle upon Tyne NE1 3BZ, United Kingdom

ENROLLING_BY_INVITATION
Hospital R&D contact (matched)

Jonathan McGregor

wcft.rdi@nhs.net0151 556 3748

How to Get in Touch

Reference Study ID Number: DSC/14/2357/51

Sponsor contact

CONTACT

+ 39 02 6443 patientadvocacy@italfarmaco.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-01