A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Recruiting Phase 4 Interventional Study
Muscular Atrophy, Spinal
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
3 Months – 24 Months
Sex
Any
Trial phase
Phase 4
Study type
Interventional
Purpose
Treatment
Participants needed
28 (estimated)
Sponsor
Hoffmann-La Roche · Industry
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About This Trial

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * \<2 years of age at the time of informed consent * Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the Survival of Motor Neuron 1 (SMN1) gene * Confirmed prese…
Contacts

Reference Study ID Number: BN44620 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