NCT07531719Recruiting
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
A Non-Interventional, Multi-Center Study Investigating the Outcomes of Risdiplam in Type I and Type II Spinal Muscle Atrophy Patients
Source: ClinicalTrials.gov · Last updated 2 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 30 (estimated)
- First submitted
- 8 Apr 2026
- Start date
- 22 Jul 2026
- Primary completion date
- 1 Mar 2028
- Lead sponsor
- Hoffmann-La Roche
- NCT number
- NCT07531719
What you commit to
- Study type: Observational
- Planned participants: 30
- From 22 Jul 2026 to the primary completion date, 1 Mar 2028
- 2 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Other illness
- Unstable gastrointestinal, renal, hepatic, endocrine or cardiovascular system diseases 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.
Other
- Participants requiring invasive ventilation or tracheostomy at index date.
- Severe contractures of joints of lower limbs and upper limbs at the time of index date.
Who the study is looking for
- 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.
Age in the registry: - to 5 years · Sex: All · Does not accept healthy volunteers
Sites (2)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Air Force Specialized Hospital Coordinator of this site? Confirm its status | Cairo | Egypt | Recruiting |
| Neurology Department, Ain Shams University Hospitals Coordinator of this site? Confirm its status | Cairo | Egypt | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.