A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Condition(s) studied
Investigational drug(s) / intervention(s)
RO7204239: RO7204239 will administered every 4 weeks (Q4W) by subcutaneous (SC) injection into the abdomen. RO7204239 will be investigated at low- and high-dose in Part 1.
Placebo: Placebo will be administered Q4W by SC injection into the abdomen.
Risdiplam: Risdiplam will be administered orally once daily (QD) for the duration of the study.
Study summary
Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which may improve how well muscles work in people with SMA. RO7204239 is an investigational anti-myostatin antibody that is designed to target myostatin. Myostatin plays an important role in the regulation of skeletal muscle size by controlling growth. Inhibiting myostatin may help muscles grow in size and strength. RO7204239 in combination with risdiplam, which is designed to increase the amount of SMN protein throughout the body, has the potential to further improve motor function and clinical outcomes for people living with SMA.
This trial will study the safety and efficacy of RO7204239 in combination with risdiplam in patients with spinal muscular atrophy (SMA). The trial has two parts; Part 1 is the dose-finding part in SMA patients that are either ambulant (aged 2-10 years) or non-ambulant (aged 5-10 years) within separate cohorts, and Part 2 is the pivotal part in SMA patients aged 2-25 years that are ambulant.
Eligibility
Primary outcome measure(s)
- Part 1 - Percentage of participants with adverse events (AEs) — Up to 4.5 years
- Part 1 - Incidence of relevant echocardiographic parameter z scores > 2 — Up to 4.5 years
- Part 1 - Serum concentration of RO7204239 — Through Week 96
- Part 1 - Time to maximum serum concentration (Cmax) of RO7204239 — Through Week 96
- Part 1 - Area under the curve (AUC) of RO7204239 — Through Week 96
- Part 1 - Trough concentration (Ctrough) of RO7204239 — Through Week 96
- Part 1 - Plasma concentration of risdiplam — Week 21
- Part 1 - Plasma concentration of risdiplam metabolite (M1) — Week 21
- Part 1 - Cmax of risdiplam — Week 21
- Part 1 - AUC of risdiplam — Week 21
- Part 1 - Ctrough of risdiplam — Week 21
- Part 1 - Incidence of anti-drug antibodies (ADAs) — Through Week 96
- Part 1 - Change from baseline in serum concentration of total myostatin — Through Week 85
- Part 1 - Change from baseline in serum concentration of free latent myostatin — Through Week 85
- Part 1 - Change from baseline in serum concentration of mature myostatin — Through Week 85
- Part 1 - Percent change from baseline in the contractile area of skeletal muscle in the dominant thigh muscles as assessed by magnetic resonance imaging (MRI) in participants aged at least 5 years — Week 24 of combination treatment
- Part 1 - Percent change from baseline in the contractile area of skeletal muscle in the dominant calf muscles as assessed by MRI in participants aged at least 5 years — Week 24 of combination treatment
- Part 2 - Change from baseline in Revised Hammersmith Scale (RHS) total score — Week 72 of combination treatment (study Week 80)
Trial sites (30)
| Facility | City | Region | Status |
|---|---|---|---|
| Boston Childrens Hospital | Boston | Massachusetts | |
| Columbia University Medical Center | New York | New York | |
| Sydney Children's Hospital | Randwick | New South Wales | |
| UZ Gent | Ghent | Belgium | |
| Chr de La Citadelle | Liège | Belgium | |
| British Columbia Children's Hospital | Vancouver | British Columbia | |
| The Hospital for Sick Children | Toronto | Ontario | |
| McGill University Health Centre - Glen Site | Montreal | Quebec | |
| Clinical Hospital Centre Zagreb | Zagreb | Croatia | |
| Policlinico Agostino Gemelli | Rome | Lazio | |
| IRCCS Istituto Giannina Gaslini | Genoa | Liguria | |
| Fondazione IRCCS Istituto Neurologico ?Carlo Besta? | Milan | Lombardy | |
| Asst Grande Ospedale Metropolitano Niguarda | Milan | Lombardy | |
| Ospedali Riuniti Torrette di Ancona | Ancona | The Marches | |
| Kobe University Hospital | Hyōgo | Japan | |
| Kagoshima University Hospital | Kagoshima | Japan | |
| National Center for Global Health and Medicine | Shinjuku-ku | Japan | |
| Universitair Medisch Centrum Utrecht | Utrecht | Netherlands | |
| Instytut Centrum Zdrowia Matki Polki | ?ód? | Poland | |
| Uniwersyteckie Centrum Kliniczne | Gda?sk | Poland | |
| Uniwersytecki Szpital Kliniczny w Poznaniu | Późna | Poland | |
| Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie | Warsaw | Poland | |
| Instytut Pomnik Centrum Zdrowia Dziecka | Warsaw | Poland | |
| CHULC, E.P.E. - Hospital Dona Estefania | Lisbon | Portugal | |
| Hospital de Santa Maria | Lisbon | Portugal | |
| Hospital Sant Joan De Deu | Esplugues de Llobregas | Barcelona | |
| Hospital Universitario La Paz | Madrid | Spain | |
| Hospital Universitario la Fe | Valencia | Spain | |
| Birmingham Heartlands Hospital | Birmingham | United Kingdom | |
| John Radcliffe Hospital | Oxford | United Kingdom |
On this site
📄 Evrysdi (risdiplam) drug profile →More Hoffmann-La Roche trials in Spain
Other trials for the same condition
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 NCT05115110 on ClinicalTrials.gov ↗ ← All trials in Spain