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Looking for participantsPhase2

Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old

Sponsor: Italfarmaco

NCT ID: NCT06769633

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 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).

Duchenne Muscular Dystrophy

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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: 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
Inclusion Criteria - Core Phase: 1. Male children aged ≥2 to \<6 years at screening (subjects ≥6 years of age at screening will not be enrolled into the study) 2. Written consent provided by parent/legal guardian and subject written assent, if applicable (according to local regulation) 3. A genetic diagnosis of DMD 4. Corticosteroid treatment considerations: 1. 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 2. For subjects without current corticosteroid treatment: Must not start corticosteroids in the Core Phase of the study (ie, first 48 weeks). Inclusion Criteria - Extension Phase: 1. Must have participated in the Core Phase study (48 weeks) and have attended the End of Treatment Visit 2. Give informed consent and /or assent in writing signed by the parent/legal guardian and/or subject (according to local regulation) 3. 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. Exclusion Criteria - Core Phase 1. Exposure to another investigational drug within 3 months prior to the start of the study drug 2. Exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study drug 3. Received any gene therapy (eg, AAV Micro-dystrophin delivery) within 12 months prior to start of study drug 4. 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. 5. 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 6. 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 7. 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 8. 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) 9. 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 10. 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) 11. 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) 12. Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening 13. 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) 14. 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 15. Hypersensitivity to any component of study drug 16. Sorbitol intolerance or malabsorption or have the hereditary form of fructose intolerance. 17. Body weight \<10 kg at screening. Exclusion Criteria - Extension Phase 1. 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) 2. 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 3. 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) 4. 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) 5. 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 6. 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.

Where Is This Study? (4 UK sites)

Leeds Teaching Hospital NHS Trust

Leeds, United Kingdom

NOT_YET_RECRUITING
Site contact (verified)
Anne-Marie Childs, MDanne-marie.childs@nhs.net

Great Ormond Street Hospital - GOSH

London, United Kingdom

NOT_YET_RECRUITING
Site contact (verified)

Newcastle upon Tyne Hospitals NHS Foundation Trust - Newcastle University

Newcastle upon Tyne NE1 eBZ, United Kingdom

Recruiting
Site contact (verified)

Oxford University Hospitals NHS Foundation Trust

Oxford OX3 9DU, United Kingdom

Recruiting
Site contact (verified)

How to Get in Touch

Italfarmaco Patient Advocacy

Sponsor contact

CONTACT

+39 02 64431 patientadvocacy@italfarmacogroup.com

Italfarmaco Patient Advocacy

Sponsor contact

CONTACT

0264431 patientadvocacy@italfarmacgroup.com
Data sourced from ClinicalTrials.gov · Last verified: 2025-07