A Phase 4 interventional study of Risdiplam in Muscular Atrophy, Spinal, sponsored by Hoffmann-La Roche. Active, not recruiting at 17 sites in 6 countries. Open to participants aged 3 Months to 24 Months. Per ClinicalTrials.gov, last updated 2026-10-22.
Sponsored by Hoffmann-La Roche · Phase 4, Interventional, and Treatment
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Among 13 studies testing risdiplam in Muscular Atrophy, Spinal — none open to participants now, 4 under way, none of them phase 3. Risdiplam · Muscular Atrophy, Spinal.
Among the 13 studies testing risdiplam in Muscular Atrophy, Spinal, this one is the 11th largest by enrollment, is one of 3 that started in 2024, is one of 4 in phase 4, and was the 12th registered.
282 studies on the registry are indexed under Muscular Atrophy, Spinal; 72 are open to participants now.
Browse Muscular Atrophy, Spinal studies →None of the 13 studies testing risdiplam in Muscular Atrophy, Spinal is open now. The open studies under Muscular Atrophy, Spinal, anywhere →
Risdiplam: first FDA approval 2020 (EVRYSDI), first EMA approval 2021. What it is approved for, and how studies give it, is on its page →
Of the 57 Muscular Atrophy, Spinal studies recruiting now, 28 (49%) set no upper age limit and 26 (46%) stop at 75 or younger; 45 take children under 18; 11 take healthy volunteers.
This study: 3 Months to 24 Months · all sexes · no healthy volunteers.
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13 studies; the count open now beside each. This study's row is marked.
First in 2016; this study’s year is marked. 3 started in 2024.
This study's enrollment of 19 is below the median of 29 across 156 interventional studies indexed under Muscular Atrophy, Spinal.
Median enrollment 33 across the 13 interventional studies. Of the 9 that completed or were terminated, 5 (56%) have results posted.
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12 of 13 have an industry lead sponsor. The 3 most frequent:
Hoffmann-La Roche is the lead sponsor of 2,101 studies on the registry; 88 are open to participants now.
Of its 330 completed or terminated interventional studies of FDA-regulated products, 248 (75%) have results posted.
18 studies in Muscular Atrophy, Spinal by Hoffmann-La Roche: 1 open now, 4 under way, none of them phase 3. Of 13 finished, 6 (46%) have results posted.
Among the 18 studies in Muscular Atrophy, Spinal by Hoffmann-La Roche, this one is the 13th largest by enrollment of the 15 interventional ones that give a count, is one of 3 that started in 2024, is one of 3 in phase 4, and was the 16th registered.
Hoffmann-La Roche’s Muscular Atrophy, Spinal studies by the year they started, since 2013.
None of the studies under way reaches primary completion within a year.
Among the 13 studies testing risdiplam in Muscular Atrophy, Spinal, from their registry updates:
Among Hoffmann-La Roche’s 18 in Muscular Atrophy, Spinal:
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This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive risdiplam orally once daily for 72 weeks (Treatment Period). The Treatment Period will be followed by a 1-year Treatment Extension Period for a total study duration of 120 weeks (approximately 2.5 years) for each participant enrolled.
Drug: Risdiplam
Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.
Also known as: RO7034067
Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment
The BSID-III is a standardized assessment commonly used to evaluate developmental functioning of infants and young children between 1 month and 42 months of age. The gross motor scale measures the movement of the limbs and torso. Items assess static positioning (e.g., sitting, standing); dynamic movement, including locomotion and coordination; balance; and motor planning. The gross motor scale consists of 72 items scored at 0 (unable to perform) or 1 (criteria for item achieved). A higher raw score indicates improvement.
Time frame: Baseline, Week 72
Percentage of Participants With Adverse Events
Time frame: Up to 120 weeks
Percentage of Participants With Serious Adverse Events
Time frame: Up to 120 weeks
Percentage of Participants With Treatment Discontinuation Due to Adverse Events
Time frame: Up to 120 weeks
Change from Baseline in Bulbar/Swallowing Function Assessment as Measured by the Oral and Swallowing Abilities Tool (OrSAT) at 72 Weeks of Risdiplam Treatment and Over Time
The OrSAT is a validated assessment composed of a checklist of 12 questions assessing aspects of swallowing abilities thought to be clinically meaningful for a type 1 SMA population and developmentally appropriate for infants during the first months of life. Each item is graded with a score of 0 or 1, depending on the child's ability to perform the activity. As some items are age-dependent, the number of items to be used, and therefore the maximum score, changes with increasing age. In the infants younger than 6 months the maximum score is 7. For those between 6 and 9 months, maximum score is 10. For infants of 10 months or older, maximum total score is 12.
Time frame: From baseline up to Week 120
Change in Swallowing Function Assessment as Measured by the Pediatric Functional Oral Intake Scale (p-FOIS) Over Time
The p-FOIS is a 6-point scale that assesses feeding ability, 1= Nothing by mouth, 2= Tube dependent for all nutrition/hydration needs with minimal attempts at oral intake for experience and/or pleasure, 3= Tube dependent with consistent intake of food and/or fluid that meets some of the nutrition/hydration needs, 4= Total oral intake but special preparation required, e.g., thickened fluids, puréed diet (where not age-appropriate), 5= Total oral intake but requiring special conditions/modification, e.g., slow flow teat/side lying/pacing or specific food limitations, 6= Total, age-appropriate, oral intake with no restrictions. Higher score indicates higher level of function.
Time frame: From baseline up to Week 120
Change from Baseline in the Raw Score of BSID-III Gross Motor Score Over Time Under Risdiplam Treatment
The BSID-III is a standardized assessment commonly used to evaluate developmental functioning of infants and young children between 1 month and 42 months of age. The gross motor scale measures the movement of the limbs and torso. Items assess static positioning (e.g., sitting, standing); dynamic movement, including locomotion and coordination; balance; and motor planning. The gross motor scale consists of 72 items scored at 0 (unable to perform) or 1 (criteria for item achieved). A higher raw score indicates improvement.
Time frame: From baseline up to Week 120
Percentage of Participants With a Gross Motor Index Between 80-109 as Measured by the Peabody Developmental Motor Scale, Third Edition (PDMS-3) at 72 Weeks of Risdiplam Treatment and Over Time
The PDMS-3 is used to assess gross- and fine-motor skills in children from birth to 5 years. The PDMS-3 has 3 composite scores: Total Motor (combined scores of the core subtests) and two domain composites (Gross Motor and Fine Motor). By combining the results of subtests, these composite scores are considered to have stronger and better indexes of performance and, therefore, more reliable and valid than the subtests. Score for composite indexes range from \<70 (impaired or delayed) to \>129 (gifted or very advanced). Score of 80-89 indicate below average and 90-109 indicate average skills.
Time frame: From baseline up to Week 120
Percentage of Participants With a Fine Motor Index Between 80-109 as Measured by the PDMS-3 at 72 Weeks of Risdiplam Treatment and Over Time
The PDMS-3 is used to assess gross- and fine-motor skills in children from birth to 5 years. The PDMS-3 has 3 composite scores: Total Motor (combined scores of the core subtests) and two domain composites (Gross Motor and Fine Motor). By combining the results of subtests, these composite scores are considered to have stronger and better indexes of performance and, therefore, more reliable and valid than the subtests. Score for composite indexes range from \<70 (impaired or delayed) to \>129 (gifted or very advanced). Score of 80-89 indicate below average and 90-109 indicate average skills.
Time frame: From baseline up to Week 120
Change in World Health Organization (WHO) Motor Milestone Achievement at 72 Weeks of Risdiplam Treatment and Over Time
The WHO motor milestones evaluate gross motor development and comprise the time windows of achievement for six gross motor milestones based on data derived from the WHO Multicenter Growth Reference Study (MGRS). The six gross motor milestones are as follows: sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone. A yes response indicates that the participant reached a particular development milestone.
Time frame: From baseline up to Week 120
Percentage of Participants With Improvement or No Change in Respiratory Illness as Assessed by Clinical Global Impression of Change (CGI-C)
In a case of respiratory illness during the study, a range of clinical domain level items will be completed by the investigator, as needed. The CGI-C is a single item measure of change using seven response options: "Very much improved," "Much improved," "Minimally improved," "No change," "Minimally worse," "Much worse," and "Very much worse."
Time frame: As per respiratory event on Day 10 and Day 20 postevent (up to Week 120)
Percentage of Participants Within 3rd Percentile of Normal Range for Weight-to-Age at 72 Weeks of Risdiplam Treatment and Over Time
Time frame: From baseline up to Week 120
Percentage of Participants Within 3rd Percentile of Normal Range for Length/Height-to-Age at 72 Weeks of Risdiplam Treatment and Over Time
Time frame: From baseline up to Week 120
Percentage of Participants Within 3rd Percentile of Normal Range for Weight-to-Length/Height at 72 Weeks of Risdiplam Treatment and Over Time
Time frame: From baseline up to Week 120
Number of Respiratory-Related Hospitalizations During the 72-Week Risdiplam Treatment and Over Time
Time frame: Up to 120 weeks
Plan to share: No — This clinical trial evaluates treatment of a rare genetic disease in a small cohort of participants. No data is planned to be shared in order to protect and maintain participant privacy/confidentiality.
Started Aug 2024 (actual). Primary completion is estimated for Mar 2028, 43 months after the start.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Risdiplam