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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At a Glance
Age
Up to 5
Sex
Any
Study type
Observational
Participants needed
30 (estimated)
Sponsor
Hoffmann-La Roche · Industry
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About This Trial
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…
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…
Contacts

Reference Study ID Number: ML45655 https://forpatients.roche.com/ No attachments to email below.

888-662-6728 (U.S. Only)

global-roche-genentech-trials@gene.com

CONTACT

Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry

CONTACT