A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
Recruiting
Observational Study
Spinal Muscle Atrophy
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
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Review the details below, then apply to join this clinical trial.
At a Glance
Age
Up to 5
Sex
Any
Study type
Observational
Participants needed
30 (estimated)
Sponsor
Hoffmann-La Roche · Industry
Who this trial is looking for
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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 vis…
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.
Trial Locations
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Eligibility Criteria
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 ind…
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.
Contacts
Reference Study ID Number: ML45655 https://forpatients.roche.com/ No attachments to email below.
Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry
CONTACT
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