Actively Recruiting
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
Led by Italfarmaco · Updated on 2026-01-21
206
Participants Needed
39
Research Sites
631 weeks
Total Duration
On this page
Sponsors
I
Italfarmaco
Lead Sponsor
C
Cromsource
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Must have participated in a previous GIVINOSTAT study in DMD and attended the End of Study Visit or been screened in study DSC/14/2357/48 meeting all inclusion and no exclusion criteria
- Baseline vastus lateralis muscle fat fraction assessed by MRS in the range 6045% or >30% (off-target group) and never randomized due to completed enrollment
- Aged 6 years or older
- Able to give informed assent and/or consent signed by the subject and/or parent/legal guardian
- Willing to use adequate contraception from previous study through 3 months after last dose, including true abstinence or appropriate contraceptive methods
You will not qualify if you...
- Use of any pharmacologic treatment other than corticosteroids affecting muscle strength/function within 3 months prior to enrollment
- Use of any investigational drug other than GIVINOSTAT
- Presence of other clinically significant diseases affecting safety or study completion
- Diagnosis of uncontrolled neurological diseases or relevant uncontrolled somatic disorders unrelated to DMD
- Platelets count, white blood cell count, or hemoglobin below lower limit of normal at screening (confirmed by repeat test)
- Fasting triglycerides over 300 mg/dL at screening (confirmed by repeat test)
- Renal impairment defined by serum Cystatin C > 2 times upper limit of normal at screening (confirmed by repeat test)
- Heart failure classified as New York Heart Association Class III or IV
- Current liver disease or impairment including elevated bilirubin > 1.5 times upper limit of normal unless due to Gilbert disease
- Baseline QTcF > 450 msec or history of risk factors for torsades de pointes
- Psychiatric illness or social situations preventing understanding or compliance with study procedures
- Hypersensitivity to study medication components
- Sorbitol intolerance, malabsorption, or hereditary fructose intolerance
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 39 locations
1
University of California - Davis Medical Center - Devis Physical Medicine & Rehabilitation
Sacramento, California, United States, 95817
Completed
2
Rady Children's Hospital center - UCSD Department of Neuroscience
San Diego, California, United States, 92123
Completed
3
Connecticut Children's Medical Center, Neurology Division
Hartford, Connecticut, United States, 06106
Completed
4
Child Health Research Institute
Gainesville, Florida, United States, 32610
Completed
5
MD Rare Disease Research, LLC
Atlanta, Georgia, United States, 30318
Enrolling by Invitation
6
University of Iowa Children's Hospital
Iowa City, Iowa, United States, 52242
Enrolling by Invitation
7
Washington University School of Medicine in St Louis Department of Neurology 660 S.Euclid Avenue, Campus Box 8111
St Louis, Missouri, United States, 63110
Completed
8
Shriners Hospitals for Children
Portland, Oregon, United States, 97239
Completed
9
The Children's Hospital of Philadelphia Colket Translational Research Building
Philadelphia, Pennsylvania, United States, 19104
Completed
10
Virginia Commonwealth University Childrens Hospital of Richmond at
Richmond, Virginia, United States, 23298
Completed
11
University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology
Leuven, Belgium, 03000
Enrolling by Invitation
12
Hospital de La Citadelle, Centre de Référence des Maladies Neuromuscolaires (CRMN)
Liège, Belgium, 04000
Enrolling by Invitation
13
Kinsmen Research Centre - Alberta Children's Hospital
Calgary, Alberta, Canada, T3B 6A8
Enrolling by Invitation
14
The University of British Columbia, Children's and Womens Health Centre of BC Branch
Vancouver, British Columbia, Canada, V6H 3V4
Enrolling by Invitation
15
Holland Bloorview Kids Rehabilitation Hospital
Toronto, Ontario, Canada, M4G1R8
Enrolling by Invitation
16
CHU de Nantes - Hotel-Dieu - Hopital Nord Laennec, rez-de-chausse haut ail Ouest
Nantes, France, 44093
Enrolling by Invitation
17
Hôpital Armand Trousseau I-Motion - Plateforme d'essais cliniques pédiatriques Bâtiment Lemariey - Porte 20 * 2ème étage 26 Avenue du Dr Arnold Nette
Paris, France, 760
Enrolling by Invitation
18
Universitätsklinikum Essen - Kinder-und Jugendmedizin Neuropadiatrie
Essen, Germany, D-45147
Enrolling by Invitation
19
Klinik- und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum HamburgEppendorf, Martinistr. 52
Hamburg, Germany, 20246
Enrolling by Invitation
20
Klinikum der Universitat Munchen, Campus Innenstadt, Lindwurmstr. 4
München, Germany, 80337
Active, Not Recruiting
21
Institute of Neurology - Schneider Children's Medical Center of Israel Kaplan, 14
Petah Tikva, Israel, 4920235
Enrolling by Invitation
22
U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative, Building 16 - ground floor IRCCS Istituto Giannina Gaslini,
Genova, Italy, 16147
Actively Recruiting
23
A.O.U. Policlinico G. Martino, U.O.C. Neurologia e Malattie Neuromuscolari
Messina, Italy, 98125
Enrolling by Invitation
24
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, UOS di Neurologia Pediatrica
Milan, Italy, 20122
Active, Not Recruiting
25
IRCCS Istituto Neurologico Carlo Besta
Milan, Italy, 20133
Enrolling by Invitation
26
Centro Clinico NeMO Fondazione Serena ONLUS Area SUD
Milan, Italy, 20162
Enrolling by Invitation
27
Ospedale Pediatrico Bambino Gesù, Malattie Neuromuscolari e Neurodegenerative
Roma, Italy, 00146
Actively Recruiting
28
Fondazione Policlinico Universitario "A.Gemelli", UOC Neuropsichiatria Infantile
Roma, Italy, 00168
Enrolling by Invitation
29
Leiden University Medical Center LUMC, Albinusdreef 2
Leiden, Netherlands, 2300
Enrolling by Invitation
30
Radboud University Medical Centre
Nijmegen, Netherlands, 6500
Enrolling by Invitation
31
Clinic of Neurology and Psychiatry for Children and Youth - Neurology Department Dr. Subotic 6a,
Belgrade, Serbia, 11000
Enrolling by Invitation
32
Neuromuscular Pathology Unit - Hospital Sant Joan de Déu
Esplugues de Llobregat, Barcellona, Spain, 08950
Enrolling by Invitation
33
Hospital Materno-Infantil
Barcelona, Spain, 08035
Actively Recruiting
34
Hospital Universitario Virgen del Rocio
Seville, Spain, 41013
Enrolling by Invitation
35
Hospital Universitari i Politècnic La Fe - Servicio Neurologia
Valencia, Spain
Enrolling by Invitation
36
Alder Hey Children's Hospital NHS Trust
Liverpool, UK, United Kingdom, L12 2AP
Enrolling by Invitation
37
The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust
Gobowen, United Kingdom, SY10 7AG
Enrolling by Invitation
38
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD
London, United Kingdom, , WC1N 1EH
Enrolling by Invitation
39
The John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, United Kingdom, NE1 3BZ
Enrolling by Invitation
Research Team
R
Reference Study ID Number: DSC/14/2357/51
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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