An upcoming study will evaluate the pharmacokinetics and safety profile of risdiplam in patients with spinal muscular atrophy (SMA).

Researchers will soon be initiating the recruiting process for this interventional study. This phase 4 clinical trial is part of an open-label study analyzing risdiplam. It will include approximately 10 participants.

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All individuals will form part of a single arm in which the intervention consists of a fixed dose of 0.15 mg/kg once a day for 28 days. The treatment will be administered orally.

The authors will identify and report the plasma concentrations, the area under the plasma concentration time curve, steady-state concentration, and free fraction of risdiplam from day 1 to day 28 as the primary outcomes regarding pharmacokinetics.

Primary outcomes that evaluate the safety of this treatment include the percentages of patients with adverse events, serious adverse events, and treatment discontinuation due to adverse events up to 30 days after the administration of the final dose.

Newborns up to 19 days of age of both sexes with a confirmed diagnosis of SMA can enroll in the study. The patients can receive a diagnosis of SMA by newborn screening or prenatal testing. All participants must be born with a gestational age of 37 weeks or more, should receive adequate hydration and nutrition when undergoing the screening test, and must be well and have recovered from any acute disease processes. Importantly, all parents and caregivers must be willing to consider feeding tube placement if the researchers recommend it.

Infants with SMA type 0, inadequate blood access, or cardiovascular abnormalities, or who have received certain medications will not be able to participate.

This novel research, titled “A phase 4, open-label study to investigate the pharmacokinetics and safety of risdiplam in infants with spinal muscular atrophy,” aims to start on June 1, 2023, and is expected to be completed by October 1, 2023.


A study to investigate the pharmacokinetics and safety of risdiplam in infants with spinal muscular atrophy (Pupfish). April 11, 2023. Accessed April 20, 2023.