New drug combo aims to boost muscle strength in SMA patients
NCT ID NCT05115110
First seen Nov 01, 2025 · Last updated Jun 23, 2026 · Updated 32 times
Summary
This study tests whether adding an experimental drug (RO7204239) that blocks myostatin—a protein that limits muscle growth—to the existing SMA medicine risdiplam (Evrysdi) can improve muscle strength and motor function. The trial includes 259 participants aged 2 to 25 with spinal muscular atrophy who can walk. Part 1 finds the right dose, and Part 2 measures how well the combo works compared to placebo plus risdiplam.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for SPINAL MUSCULAR ATROPHY (SMA) are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
-
Asst Grande Ospedale Metropolitano Niguarda
Milan, Lombardy, 20162, Italy
-
Birmingham Heartlands Hospital
Birmingham, B9 5SS, United Kingdom
-
Boston Childrens Hospital
Boston, Massachusetts, 02115, United States
-
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3N1, Canada
-
CHULC, E.P.E. - Hospital Dona Estefania
Lisbon, 1169-045, Portugal
-
Chr de La Citadelle
Liège, 4000, Belgium
-
Clinical Hospital Centre Zagreb
Zagreb, 10000, Croatia
-
Columbia University Medical Center
New York, New York, 10032, United States
-
Fondazione IRCCS Istituto Neurologico ?Carlo Besta?
Milan, Lombardy, 20133, Italy
-
Great Ormond Street Hospital For Children
London, WC1N 3JH, United Kingdom
-
Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, 08950, Spain
-
Hospital Universitario La Paz
Madrid, 28046, Spain
-
Hospital Universitario la Fe
Valencia, 46026, Spain
-
Hospital Vall d'Hebron
Barcelona, 08035, Spain
-
Hospital de Santa Maria
Lisbon, 1649-035, Portugal
-
IRCCS Istituto Giannina Gaslini
Genoa, Liguria, 16147, Italy
-
Instytut Centrum Zdrowia Matki Polki
?ód?, 93-338, Poland
-
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, 04-730, Poland
-
John Radcliffe Hospital
Oxford, OX3 9DU, United Kingdom
-
Kagoshima University Hospital
Kagoshima, 890-8520, Japan
-
Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie
Warsaw, 02-097, Poland
-
Kobe University Hospital
Hyōgo, 650-0017, Japan
-
McGill University Health Centre - Glen Site
Montreal, Quebec, H4A 3J1, Canada
-
National Center for Global Health and Medicine
Shinjuku-ku, 162-8655, Japan
-
Nemours Children's Hospital
Orlando, Florida, 32827, United States
-
Neurology & Neuromuscular Care Center
Flower Mound, Texas, 75028, United States
-
Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 00165, Italy
-
Ospedali Riuniti Torrette di Ancona
Ancona, The Marches, 60126, Italy
-
Policlinico Agostino Gemelli
Rome, Lazio, 00168, Italy
-
Sydney Children's Hospital
Randwick, New South Wales, 2031, Australia
-
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
-
UZ Gent
Ghent, 9000, Belgium
-
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
-
Uniwersytecki Szpital Kliniczny w Poznaniu
Późna, 60-355, Poland
-
Uniwersyteckie Centrum Kliniczne
Gda?sk, 80-952, Poland
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
RO7204239 (an experimental antibody that blocks myostatin to help muscles grow) combined with risdiplam (Evrysdi, a daily oral drug that boosts SMN protein)
What this could lead to
If it works, this combination could improve muscle strength and motor function more than risdiplam alone for people with SMA.
What could go wrong
This is an early-to-mid stage trial (Phase 2/3) with only 259 participants. The experimental drug may not provide added benefit, and side effects are still being studied.
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.