A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA) (NCT07531719) | Clinical Trial Compass
Not Yet RecruitingNot Applicable
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
30 participantsStarted 2026-07-15
Plain-language summary
This multicenter, non-interventional, prospective study with a retrospective component aims to evaluate the real-world clinical outcomes, safety profile, and motor function improvements of Risdiplam treatment in participants with Type I and Type II Spinal Muscle Atrophy over 24 months of treatment.
The study will allow retrospective collection of medical history data and previous participants visits from the medical records.
Who can participate
Age range
5 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Maximum age to be 6 months for type I participants and 5 years for type II participants at the time of diagnosis.
* Participants must start/have started treatment with Risdiplam within 6 months of diagnosis.
* Participant to be receiving adequate nutrition and hydration at index date, in the opinion of the Investigator.
* Participant to be adequately recovered from any acute illness at the time index date.
Exclusion Criteria:
* Participants not receiving treatment for disease with Risdiplam according to standard of care and in line with the current summary of product characteristics (SPC)/local labeling.
* Participants not receiving the Roche studied medicinal product, but a biosimilar.
* Concomitant or previous administration in any investigational drug or device study.
* Concomitant or previous administration of approved treatment for SMA other than Risdiplam.
* Unstable gastrointestinal, renal, hepatic, endocrine or cardiovascular system diseases at index date.
* Participants requiring invasive ventilation or tracheostomy at index date.
* Participants requiring awake non-invasive ventilation due to respiratory insufficiency or with awake hypoxemia with or without ventilator support or with history of respiratory failure or severe pneumonia, and have not fully recovered their pulmonary function at the time of index date.
* Severe contractures of joints of lower limbs and upper limbs at the time of index date.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change From Index Date in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score at Month 12
Timeframe: Baseline, Month 12
2
Change From Index Date in Hammersmith Functional Motor Scale Expanded (HFMSE) Score at Months 12
Timeframe: Baseline, Months 12
Trial details
NCT IDNCT07531719
SponsorHoffmann-La Roche
Sponsor typeINDUSTRY
Study typeOBSERVATIONAL
Primary completion2028-03-01
Contact for this trial
Reference Study ID Number: ML45655 https://forpatients.roche.com/