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Clinical Trials in the UK / NCT03373968
Recruiting Phase 2/3

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

NCT03373968 · tracked via the Priya Life Science UK tracker
Sponsor
Italfarmaco
Phase
Phase 2/3
Started
2017-10-24
Last updated
2026-01-21

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

Givinostat

Givinostat: suspension of givinostat (10 mg/mL)

Study summary

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.

Eligibility

Sex
MALE
Min age
7 Years
Max age
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (39)

FacilityCityRegionStatus
University of California - Davis Medical Center - Devis Physical Medicine & Rehabilitation Sacramento California Completed
Rady Children's Hospital center - UCSD Department of Neuroscience San Diego California Completed
Connecticut Children's Medical Center, Neurology Division Hartford Connecticut Completed
Child Health Research Institute Gainesville Florida Completed
MD Rare Disease Research, LLC Atlanta Georgia Enrolling By Invitation
University of Iowa Children's Hospital Iowa City Iowa Enrolling By Invitation
Washington University School of Medicine in St Louis Department of Neurology 660 S.Euclid Avenue, Campus Box 8111 St Louis Missouri Completed
Shriners Hospitals for Children Portland Oregon Completed
The Children's Hospital of Philadelphia Colket Translational Research Building Philadelphia Pennsylvania Completed
Virginia Commonwealth University Childrens Hospital of Richmond at Richmond Virginia Completed
University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology Leuven Belgium Enrolling By Invitation
Hospital de La Citadelle, Centre de Référence des Maladies Neuromuscolaires (CRMN) Liège Belgium Enrolling By Invitation
Kinsmen Research Centre - Alberta Children's Hospital Calgary Alberta Enrolling By Invitation
The University of British Columbia, Children's and Womens Health Centre of BC Branch Vancouver British Columbia Enrolling By Invitation
Holland Bloorview Kids Rehabilitation Hospital Toronto Ontario Enrolling By Invitation
CHU de Nantes - Hotel-Dieu - Hopital Nord Laennec, rez-de-chausse haut ail Ouest Nantes France Enrolling By Invitation
Hôpital Armand Trousseau I-Motion - Plateforme d'essais cliniques pédiatriques Bâtiment Lemariey - Porte 20 * 2ème étage 26 Avenue du Dr Arnold Nette Paris France Enrolling By Invitation
Universitätsklinikum Essen - Kinder-und Jugendmedizin Neuropadiatrie Essen Germany Enrolling By Invitation
Klinik- und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum HamburgEppendorf, Martinistr. 52 Hamburg Germany Enrolling By Invitation
Klinikum der Universitat Munchen, Campus Innenstadt, Lindwurmstr. 4 München Germany Active Not Recruiting
Institute of Neurology - Schneider Children's Medical Center of Israel Kaplan, 14 Petah Tikva Israel Enrolling By Invitation
U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative, Building 16 - ground floor IRCCS Istituto Giannina Gaslini, Genova Italy Recruiting
A.O.U. Policlinico G. Martino, U.O.C. Neurologia e Malattie Neuromuscolari Messina Italy Enrolling By Invitation
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, UOS di Neurologia Pediatrica Milan Italy Active Not Recruiting
IRCCS Istituto Neurologico Carlo Besta Milan Italy Enrolling By Invitation
Centro Clinico NeMO Fondazione Serena ONLUS Area SUD Milan Italy Enrolling By Invitation
Ospedale Pediatrico Bambino Gesù, Malattie Neuromuscolari e Neurodegenerative Roma Italy Recruiting
Fondazione Policlinico Universitario "A.Gemelli", UOC Neuropsichiatria Infantile Roma Italy Enrolling By Invitation
Leiden University Medical Center LUMC, Albinusdreef 2 Leiden Netherlands Enrolling By Invitation
Radboud University Medical Centre Nijmegen Netherlands Enrolling By Invitation
Clinic of Neurology and Psychiatry for Children and Youth - Neurology Department Dr. Subotic 6a, Belgrade Serbia Enrolling By Invitation
Neuromuscular Pathology Unit - Hospital Sant Joan de Déu Esplugues de Llobregat Barcellona Enrolling By Invitation
Hospital Materno-Infantil Barcelona Spain Recruiting
Hospital Universitario Virgen del Rocio Seville Spain Enrolling By Invitation
Hospital Universitari i Politècnic La Fe - Servicio Neurologia Valencia Spain Enrolling By Invitation
Alder Hey Children's Hospital NHS Trust Liverpool UK Enrolling By Invitation
The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust Gobowen United Kingdom Enrolling By Invitation
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD London United Kingdom Enrolling By Invitation
The John Walton Muscular Dystrophy Research Centre Newcastle upon Tyne United Kingdom Enrolling By Invitation

More Italfarmaco trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03373968 on ClinicalTrials.gov ↗ ← All trials in the UK