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DEVOTE Study Exploring Higher Dose of Nusinersen in Spinal Muscular Atrophy: Study Design and Part A Results
Richard S Finkel1, John W Day2, Samuel Ignacio Pascual Pascual3
1Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.
Journal of Neuromuscular Diseases
|July 2, 2023
Summary
The DEVOTE study
Area of Science:
- Neurology
- Pharmacology
- Clinical Trials
Background:
- Pharmacokinetic/pharmacodynamic modeling suggests higher nusinersen doses may improve efficacy.
- The approved dose of nusinersen is 12 mg.
Purpose of the Study:
- To evaluate the safety, tolerability, and efficacy of higher doses of nusinersen.
- To describe the design of the 3-part DEVOTE clinical study (NCT04089566).
Main Methods:
- Part A assessed safety and tolerability of higher nusinersen doses in pediatric participants.
- Parts B and C involve efficacy assessment and dose transition evaluations, respectively.
- The study enrolled 6 participants aged 6.1-12.6 years in Part A.
Main Results:
- Part A completed with no safety concerns; most treatment-emergent adverse events were mild and procedure-related.
- Cerebrospinal fluid nusinersen levels aligned with model predictions.
- Most participants demonstrated motor function stabilization or improvement, despite Part A not being efficacy-focused.
Conclusions:
- Part A findings support the continued development of higher nusinersen doses.
- Further investigation in Parts B and C is ongoing.
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