Clareo Health | A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
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CONTACT
Lead
Hoffmann-La Roche
Age
0–2
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
\<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 presence of two SMN2 gene copies as documented through laboratory testing
Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically
Has received onasemnogene abeparvovec for SMA no less than 13 weeks, but not more than months 30 weeks, prior to enrollment
If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration
Has, in the opinion of the investigator, not experienced clinically significant decline in function from the time of onasemnogene abeparvovec administration
You may not be if
Previous or current enrolment in investigational study prior to initiation of study treatment
Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information
Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide
Concomitant or previous use of an anti-myostatin agent
Participants requiring invasive ventilation or tracheostomy
Participants requiring awake non-invasive ventilation or with awake hypoxemia (Arterial Oxygen Saturation \[SaO2\] \<95%) with or without ventilator support
Presence of feeding tube and an OrSAT score of 0
Hospitalization for pulmonary event within the last 2 months, or any planned hospitalization at the time of screening
Any major illness requiring hospitalization within 1 month before the screening examination or any febrile illness within 1 week prior to screening and up to first dose administration.
