Actively Recruiting
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
Led by Italfarmaco · Updated on 2025-05-11
138
Participants Needed
20
Research Sites
206 weeks
Total Duration
On this page
Sponsors
I
Italfarmaco
Lead Sponsor
F
Fortrea
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety, and tolerability of givinostat in non-ambulant male paediatric (aged 9 to \<18 years) patients with DMD. 138 patients will be randomised 2:1 to givinostat or placebo and will be treated for 18 months. * Planned screening duration: approximately 4 weeks (±14 days) * Planned treatment duration: 18 months (approximately 72 weeks) * Planned follow-up duration: 4 weeks (±7 days) (for patients not participating in the long-term safety study) * Total duration of study participation: up to 83 weeks (ie, 20-21 months)
CONDITIONS
Official Title
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male children and adolescents aged 9 to under 18 years at screening
- Ability to give informed assent and/or consent by patient and/or parent/legal guardian
- Confirmed genetic diagnosis of Duchenne muscular dystrophy
- Non-ambulant status defined as wheelchair bound and unable to perform or complete 10-meter walk/run test within 30 seconds without support
- Performance of Upper Limb (PUL) 2.0 entry scores between 3 and 6
- Stable medication for DMD-associated cardiomyopathy for at least 1 month prior to treatment start if applicable
- Stable corticosteroid use for at least 6 months prior to treatment start with no significant dose changes
- Willingness to use adequate contraception from randomisation through 3 months after last dose
You will not qualify if you...
- Use of any investigational drug within 3 months before treatment start
- Exposure to dystrophin restoration products within 6 months before treatment start
- Prior gene therapy for DMD
- Use of pharmacologic treatments or supplements (other than corticosteroids) affecting muscle strength within 3 months before treatment
- Use of testosterone except stable replacement therapy for delayed puberty
- Elbow-flexion contractures greater than 30 degrees in dominant arm
- Inability to perform consistent PUL 2.0 measurements at screening
- Forced Vital Capacity less than 40% predicted
- Requirement for daytime ventilator assistance
- Respiratory failure episode within 8 weeks prior to screening
- Symptomatic cardiomyopathy or heart failure with left ventricular ejection fraction below 45%
- QTcF interval above 450 msec or risk factors for torsades de pointes
- Planned major surgery including scoliosis surgery within 1 year
- Poorly controlled asthma or lung disease impacting respiratory function
- Low platelets, white blood cells, or hemoglobin below normal limits
- Fasting triglycerides above 300 mg/dL after repeat testing
- Current or history of liver disease or impairment except Gilbert disease
- Inadequate renal function based on serum Cystatin C levels after repeat testing
- Positive tests for hepatitis B, hepatitis C, or HIV
- Hypersensitivity to study medication components
- Sorbitol intolerance, malabsorption, or hereditary fructose intolerance
- Other uncontrolled neurological or somatic disorders unrelated to DMD
- Psychiatric or social conditions preventing understanding or compliance
- Contraindications to MRI such as claustrophobia or metal implants
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 20 locations
1
Universitaire Ziekenhuizen Leuven
Leuven, Belgium, 3000
Actively Recruiting
2
British Columbia Children's Hospital
Vancouver, British Columbia, Canada, V6H 3V4
Actively Recruiting
3
The University of Western Ontario - Children's Health Research Institute
London, Ontario, Canada, N6A 5W9
Actively Recruiting
4
University of Ottawa - Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Actively Recruiting
5
University of Toronto - Holland Bloorview Kids Rehabilitation Hospital
Toronto, Ontario, Canada, M4G 1R8
Actively Recruiting
6
Centre Hospitalier Régional Universitaire de Lille
Lille, France, 59037
Actively Recruiting
7
Centre hospitalier universitaire - Hôpitaux de Marseille
Marseille, France, 13385
Actively Recruiting
8
Hôpital Armand-Trousseau - I-Motion
Paris, France, 75935
Actively Recruiting
9
Charite-Universitaetsmedizin Berlin
Berlin, Germany, 10117
Actively Recruiting
10
Universitaetsklinikum Freiburg
Freiburg im Breisgau, Germany, 53113
Actively Recruiting
11
Associazione La Nostra Famiglia - IRCCS Eugenio Medea - Bosisio Parini
Lecco, Italy, 23842
Actively Recruiting
12
Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca' Granda - NeuroMuscular Omnicentre
Milan, Italy, 20162
Actively Recruiting
13
Università degli Studi di Padova - Azienda Ospedaliera di Padova
Padova, Italy, 35128
Actively Recruiting
14
Ospedale Pediatrico Bambino Gesù
Roma, Italy, 00165
Actively Recruiting
15
Policlinico Universitario Agostino Gemelli - Università Cattolica del Sacro Cuore
Roma, Italy, 00165
Actively Recruiting
16
Leids Universitair Medisch Centrum (LUMC)
Leiden, Netherlands, 2300 RC
Actively Recruiting
17
Radboud Universitair Medisch Centrum (Radboudumc)
Nijmegen, Netherlands, 6500 HB
Actively Recruiting
18
Newcastle upon Tyne Hospitals NHS Foundation Trust - Newcastle University
Newcastle upon Tyne, England, United Kingdom, NE1 3BZ
Active, Not Recruiting
19
Oxford University Hospitals NHS Foundation Trust
Oxford, England, United Kingdom, OX3 9DU
Withdrawn
20
NHS Greater Glasgow and Clyde - Royal Hospital for Children
Glasgow, Scotland, United Kingdom, G51 4TF
Active, Not Recruiting
Research Team
I
Italfarmaco Patient Advocacy
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here