Experimental drug aims to preserve arm and lung function in boys with advanced duchenne
NCT ID NCT05933057
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for DUCHENNE MUSCULAR DYSTROPHY are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Associazione La Nostra Famiglia - IRCCS Eugenio Medea - Bosisio Parini
RECRUITINGLecco, 23842, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
British Columbia Children's Hospital
RECRUITINGVancouver, British Columbia, V6H 3V4, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Centre Hospitalier Régional Universitaire de Lille
RECRUITINGLille, 59037, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Centre hospitalier universitaire - Hôpitaux de Marseille
RECRUITINGMarseille, 13385, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Charite-Universitaetsmedizin Berlin
RECRUITINGBerlin, 10117, Germany
Contact Email: •••••@•••••
Contact
-
Clinic of Neurology and Psychiatry for Children and Youth
NOT_YET_RECRUITINGBelgrade, 11000, Serbia
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca' Granda - NeuroMuscular Omnicentre
RECRUITINGMilan, 20162, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Hospital Sant Joan de Déu
RECRUITINGBarcelona, 08950, Spain
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Hospital Universitario y Politécnico La Fe
RECRUITINGValencia, 46026, Spain
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Hôpital Armand-Trousseau - I-Motion
RECRUITINGParis, 75935, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Karolinska University Hospital, Solna CKB Centrum för Kliniska Barnstudier
NOT_YET_RECRUITINGStockholm, 17176, Sweden
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Klinika dětské neurologie 2. LF, Fakultní nemocnice v Motole
NOT_YET_RECRUITINGPrague, 15006, Czechia
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Leids Universitair Medisch Centrum (LUMC)
RECRUITINGLeiden, 2300 RC, Netherlands
Contact Email: •••••@•••••
Contact
-
NHS Greater Glasgow and Clyde - Royal Hospital for Children
ACTIVE_NOT_RECRUITINGGlasgow, Scotland, G51 4TF, United Kingdom
-
Newcastle upon Tyne Hospitals NHS Foundation Trust - Newcastle University
ACTIVE_NOT_RECRUITINGNewcastle upon Tyne, England, NE1 3BZ, United Kingdom
-
Ospedale Pediatrico Bambino Gesù
RECRUITINGRoma, 00165, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Email: •••••@•••••
Contact
-
Oxford University Hospitals NHS Foundation Trust
WITHDRAWNOxford, England, OX3 9DU, United Kingdom
-
Policlinico Universitario Agostino Gemelli - Università Cattolica del Sacro Cuore
RECRUITINGRoma, 00165, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Radboud Universitair Medisch Centrum (Radboudumc)
RECRUITINGNijmegen, 6500 HB, Netherlands
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
The University of Western Ontario - Children's Health Research Institute
RECRUITINGLondon, Ontario, N6A 5W9, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Universitaetsklinikum Freiburg
RECRUITINGFreiburg im Breisgau, 53113, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Universitaire Ziekenhuizen Leuven
RECRUITINGLeuven, 3000, Belgium
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
University of Ottawa - Children's Hospital of Eastern Ontario
RECRUITINGOttawa, Ontario, K1H 8L1, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
University of Toronto - Holland Bloorview Kids Rehabilitation Hospital
RECRUITINGToronto, Ontario, M4G 1R8, Canada
Contact Email: •••••@•••••
Contact
-
Università degli Studi di Padova - Azienda Ospedaliera di Padova
RECRUITINGPadova, 35128, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Email: •••••@•••••
Contact
-
Universitätsklinikum Essen Kinder-und Jugendmedizin Neuropadiatrie
NOT_YET_RECRUITINGEssen, 45147, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Universitätsklinikum Hamburg-Eppendorf, Klinik und Poliklinik für Kinder- und Jugendmedizin
NOT_YET_RECRUITINGHamburg, 20246, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny
NOT_YET_RECRUITINGWarsaw, 02097, Poland
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Uniwersyteckie Centrum Kliniczne, Klinika Neurologii Rozwojowej
NOT_YET_RECRUITINGGdansk, 80952, Poland
Contact Phone: •••-•••-•••• Email: •••••@•••••
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a massive patient database unlock new treatments for muscular dystrophy?
- Umbilical cord stem cells aim to slow muscle loss in duchenne boys
- Experimental drug KER-065 aims to slow muscle decline in duchenne muscular dystrophy
- Can early parent coaching help infants with rare genetic disorders thrive?
- Newborn screening study aims to catch rare diseases at birth
- One-Time gene infusion aims to fight duchenne muscular dystrophy