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Experimental drug aims to preserve arm and lung function in boys with advanced duchenne

NCT ID NCT05933057

Disease control Sponsor: Italfarmaco Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 20, 2026 · Last updated Jul 24, 2026 · Updated 2 times

Summary

This phase 3 trial tests whether givinostat, an oral drug, can slow muscle decline in boys aged 9 to 18 with Duchenne muscular dystrophy who can no longer walk. Participants receive either givinostat or a placebo for 18 months. The main goal is to see if the drug helps maintain upper arm function and breathing capacity.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
givinostat
What this could lead to
If successful, givinostat could become a treatment to slow muscle decline and preserve arm and lung function in boys with advanced Duchenne muscular dystrophy.
What could go wrong
This is a relatively small, early-phase trial, and results may not confirm benefit. Side effects or lack of efficacy could limit its use.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 138 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2024

Expected to finish

Feb 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

9 to 17 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: Patients must satisfy all the following criteria: 1. Children and adolescent males aged ≥ 9 to \<18 years at screening (patients ≥ 18 years of age at screening will not be enrolled into the study) 2. Are able to give informed assent and/or consent in writing signed by the patient and/or parent/legal guardian (according to local regulations) 3. A genetic diagnosis of DMD 4. Non-ambulant, defined as being wheelchair bound and: 1. Unable to perform the 10-meter walk/run test (10MWT), or 2. Unable to complete the 10MWT in 30 seconds or less, without any support or devices 5. Performance of the Upper Limb test (PUL version 2.0) entry item scores 3 to 6 6. If on medication for DMD-associated cardiomyopathy (eg, ACE inhibitor, β-blocker, diuretics), stable for ≥1 month immediately prior to start of study treatment, if any 7. Stable corticosteroids, defined as: 1. Receiving systemic corticosteroids for a minimum of 6 months immediately prior to start of study treatment 2. No significant change in dose or dosing regimen (except for adjustments due to body weight change) for a minimum of 6 months immediately prior to start of study treatment 8. Willing to use adequate contraception. Effective contraceptive methods must be used from randomisation visit through 3 months after the last dose of study drug, and include the following: 1. True abstinence (ie, absence of any sexual intercourse), when in line with the preferred and usual lifestyle of the patient. Periodic abstinence (eg, calendar, ovulation, post-ovulation, and symptothermal methods) and withdrawal are not acceptable methods of contraception 2. Condom with spermicide and the female partner must use an effective method of contraception, such as an oral, transdermal, injectable or implanted hormonal contraceptive; intrauterine device; bilateral tubal occlusion, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such as for example cervical cap with spermicide jelly. Exclusion Criteria: Patients will be excluded from the study if they satisfy any of the following criteria: 1. Exposure to another investigational drug within 3 months prior to start of study treatment. 2. Have exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study treatment 3. Having received any gene therapy (eg, AAV Micro-dystrophin delivery) prior to start of study treatment 4. Use of any pharmacologic treatment or supplement (other than corticosteroids), that might have had an effect on muscle strength or function within 3 months prior to the start of study treatment (eg, growth hormone); vitamin D, calcium and any other supplements will be allowed 5. Use of testosterone, unless used as a replacement therapy for the treatment of delayed puberty. The testosterone dose and regimen should be stable within 6 months prior to the start of study treatment, and circulating testosterone levels should be within the normal ranges for the patient's age 6. Elbow-flexion contractures \>30° in the dominant arm 7. Inability to perform consistent PUL 2.0 measurement within ±2 points without shoulder domain or within ±3 points with shoulder domain during paired testing at screening 8. Forced Vital Capacity % of predicted \<40% 9. Requirement for daytime ventilator assistance (Note: Night ventilator assistance and use of bi-level positive airway pressure therapy is allowed) 10. Episode of respiratory failure within the 8 weeks prior to screening 11. Symptomatic cardiomyopathy or heart failure and/or left ventricular ejection fraction \<45% 12. Baseline corrected QT interval using Fredericia's formula (QTcF) \>450 msec (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (eg, heart failure, hypokalaemia, or family history of long QT syndrome) 13. Major surgical procedure (including scoliosis surgery) planned within 1 year of the start of study treatment 14. Poorly controlled asthma or underlying lung disease such as bronchitis, bronchiectasis, emphysema, recurrent pneumonia that in the opinion of the Investigator might impact respiratory function 15. Platelets, white blood cells, and/or haemoglobin \< lower limit of normal (LLN) at screening (Note: for abnormal screening laboratory test results \[\<LLN\], the platelets count, white blood cell, and haemoglobin will be repeated once; if the repeat test result is still \<LLN, the patient should be excluded) 16. 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, the patient should be excluded) 17. 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 18. 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 patient should be excluded) 19. Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening 20. Hypersensitivity to any component of study medication 21. Sorbitol intolerance or malabsorption, or have the hereditary form of fructose intolerance 22. Diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD, based on Investigator judgement 23. Psychiatric illness or social situations rendering the potential patient unable to understand and comply with the muscle function tests and/or with the study protocol procedures, based on Investigator judgement 24. Have contraindications to MRI scan (eg, claustrophobia, metal implants, or uncontrolled seizure disorder), based on Investigator's judgement.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Duchenne muscular dystrophy

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    26 sites in 11 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Associazione La Nostra Famiglia - IRCCS Eugenio Medea - Bosisio Parini

    RECRUITING

    Lecco, 23842, Italy

  • British Columbia Children's Hospital

    RECRUITING

    Vancouver, British Columbia, V6H 3V4, Canada

  • Centre Hospitalier Régional Universitaire de Lille

    RECRUITING

    Lille, 59037, France

  • Centre hospitalier universitaire - Hôpitaux de Marseille

    RECRUITING

    Marseille, 13385, France

  • Charite-Universitaetsmedizin Berlin

    RECRUITING

    Berlin, 10117, Germany

  • Clinic of Neurology and Psychiatry for Children and Youth

    NOT_YET_RECRUITING

    Belgrade, 11000, Serbia

  • Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca' Granda - NeuroMuscular Omnicentre

    RECRUITING

    Milan, 20162, Italy

  • Hospital Sant Joan de Déu

    RECRUITING

    Barcelona, 08950, Spain

  • Hospital Universitario y Politécnico La Fe

    RECRUITING

    Valencia, 46026, Spain

  • Hôpital Armand-Trousseau - I-Motion

    RECRUITING

    Paris, 75935, France

  • Karolinska University Hospital, Solna CKB Centrum för Kliniska Barnstudier

    NOT_YET_RECRUITING

    Stockholm, 17176, Sweden

  • Klinika dětské neurologie 2. LF, Fakultní nemocnice v Motole

    NOT_YET_RECRUITING

    Prague, 15006, Czechia

  • Leids Universitair Medisch Centrum (LUMC)

    RECRUITING

    Leiden, 2300 RC, Netherlands

  • NHS Greater Glasgow and Clyde - Royal Hospital for Children

    ACTIVE_NOT_RECRUITING

    Glasgow, Scotland, G51 4TF, United Kingdom

  • Newcastle upon Tyne Hospitals NHS Foundation Trust - Newcastle University

    ACTIVE_NOT_RECRUITING

    Newcastle upon Tyne, England, NE1 3BZ, United Kingdom

  • Ospedale Pediatrico Bambino Gesù

    RECRUITING

    Roma, 00165, Italy

    Contact Email: •••••@•••••

  • Oxford University Hospitals NHS Foundation Trust

    WITHDRAWN

    Oxford, England, OX3 9DU, United Kingdom

  • Policlinico Universitario Agostino Gemelli - Università Cattolica del Sacro Cuore

    RECRUITING

    Roma, 00165, Italy

  • Radboud Universitair Medisch Centrum (Radboudumc)

    RECRUITING

    Nijmegen, 6500 HB, Netherlands

  • The University of Western Ontario - Children's Health Research Institute

    RECRUITING

    London, Ontario, N6A 5W9, Canada

  • Universitaetsklinikum Freiburg

    RECRUITING

    Freiburg im Breisgau, 53113, Germany

  • Universitaire Ziekenhuizen Leuven

    RECRUITING

    Leuven, 3000, Belgium

  • University of Ottawa - Children's Hospital of Eastern Ontario

    RECRUITING

    Ottawa, Ontario, K1H 8L1, Canada

  • University of Toronto - Holland Bloorview Kids Rehabilitation Hospital

    RECRUITING

    Toronto, Ontario, M4G 1R8, Canada

  • Università degli Studi di Padova - Azienda Ospedaliera di Padova

    RECRUITING

    Padova, 35128, Italy

  • Universitätsklinikum Essen Kinder-und Jugendmedizin Neuropadiatrie

    NOT_YET_RECRUITING

    Essen, 45147, Germany

  • Universitätsklinikum Hamburg-Eppendorf, Klinik und Poliklinik für Kinder- und Jugendmedizin

    NOT_YET_RECRUITING

    Hamburg, 20246, Germany

  • Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny

    NOT_YET_RECRUITING

    Warsaw, 02097, Poland

  • Uniwersyteckie Centrum Kliniczne, Klinika Neurologii Rozwojowej

    NOT_YET_RECRUITING

    Gdansk, 80952, Poland

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