New Muscle-Boosting drug combo tested for SMA

NCT ID NCT05115110

Ongoing Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests whether adding an experimental drug (RO7204239) to an existing SMA medicine (risdiplam) can help people with spinal muscular atrophy build stronger muscles and move better. The trial includes about 259 children and young adults, ages 2 to 25, who can walk. Researchers will check safety, side effects, and how well the combination works over time.

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) combined with risdiplam (Evrysdi)
What this could lead to: If it works, this combination could help people with SMA grow stronger muscles and improve their ability to move and walk.
What could go wrong: This is an early-to-mid-stage trial, so the added benefit of RO7204239 over risdiplam alone is not yet proven. Side effects from the new antibody are possible and being studied.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SPINAL MUSCULAR ATROPHY (SMA)

Contacts and locations

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

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