Two new clinical trials of nusinersen for spinal muscular atrophy (SMA) are now recruiting participants.

The first called ASCEND is a phase 3b study to evaluate a higher dose of the treatment in patients who have previously been treated with risdiplam. Participants will receive 2 loading doses of 50 mg nusinersen approximately 2 weeks apart, which will be followed by maintenance doses of 28 mg approximately every 4 months.

The primary objective is to evaluate motor function following treatment and the secondary objective is to evaluate the safety and tolerability of the treatment.


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ASCEND is currently recruiting an estimated 135 participants with SMA, aged 5 to 39 years in Arkansas, Georgia, North Carolina, and Virginia, and is estimated to be completed on June 14, 2027.

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The second trial called RESPOND is a phase 4 study of nusinersen in patients who were previously treated with onasemnogene abeparvovec. The primary objective is to evaluate the clinical outcomes following treatment with nusinersen, and secondary objectives include evaluating the safety and tolerability.

An estimated 60 participants with SMA aged 2 to 36 months will be given 12 mg of nusinersen intrathecally as loading doses on days 1, 15, 29, and 64. This will be followed by maintenance doses, every 4 months, on days 183, 302, 421, 540, and 659.

RESPOND is currently recruiting participants in the US, Germany, Israel, Italy, and Spain. It is expected to be completed on September 4, 2024.

Nusinersen is an antisense oligonucleotide developed by Biogen to treat children and adults with SMA. It works by correcting exon 7 splicing in the SMN2 gene and increasing the amount of SMN protein made from this gene. It has been approved by the US Food and Drug Administration on December 23, 2016, and the European Medicines Agency in June of 2017.

References

A study to evaluate higher dose (HD) nusinersen (BIIB058) in participants with spinal muscular atrophy previously treated with risdiplam (ASCEND). US National Library of Medicine. Updated March 10, 2022. Accessed March 22, 2022. 

A study of nusinersen among participants with spinal muscular atrophy who received onasemnogene abeparvovec (RESPOND). US National Library of Medicine. Updated March 7, 2022. Accessed March 22, 2022.