Risdiplam in Type I and Type II Spinal Muscular Atrophy: real-world outcomes

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a multicenter, non-interventional study with a retrospective component designed to observe the real-world use of risdiplam in SMA patients. It will collect medical history and prior visit data from records, and follow participants for up to 24 months to monitor motor function and safety. The study includes participants who started risdiplam within six months of diagnosis and are in good nutritional and health status at the index date. Data will come from clinical records at participating sites in Cairo, Egypt. No interventions beyond standard risdiplam treatment are administered within this observational framework.

Why it matters

Who can join: the full rules

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.

Where it's running

• Neurology Department, Ain Shams University Hospitals
  Cairo, Egypt Recruiting
• Air Force Specialized Hospital
  Cairo, Egypt Recruiting

Study contacts

• Study coordinator: Reference Study ID Number: ML45655 https://forpatients.roche.com/ No attachments to email below.
• Email: global-roche-genentech-trials@gene.com
• Phone: 888-662-6728 (U.S. Only)

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Apitegromab for infants under 2 with spinal muscular atrophy

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.