A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy

A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen

Status

Active, not recruiting

Phases

Phase 4

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2031260173

Enrollment

172

Registered

2026-06-17

Start date

2022-08-17

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Spinal muscular atrophy

Interventions

Research Name: BIIB058 Generic Name: nusinersen Trade Names: Spinraza Participants in Study 203 who received maintenance doses of 28 mg nusinersen every 4 months will continue this dosing scheme in S

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Completion of the Day 302 Visit in Study 203 and compliance with the study protocol throughout Study 203.

Exclusion criteria

Exclusion criteria: -Treatment with another investigational therapy or enrollment in another interventional clinical study. -Treatment with an approved therapy for SMA after the Day 302 Visit of Study 203.F68

Design outcomes

Primary

Measure	Time frame
-Incidence of AEs, including SAE -Change in growth parameters -Shifts from baseline in clinical laboratory parameters, ECGs, and vital signs -Shifts from baseline in coagulation parameters (aPTT, PT, and INR) -Change in urine total protein -Change from baseline in neurological examination outcomes -The proportion of participants with a postbaseline platelet count below the lower limit of normal on at least 2 consecutive measurements -The proportion of participants with a postbaseline QTcF of > 500 msec and an increase from baseline to any postbaseline timepoint in QTcF of > 60 msec	—

Measure

Time frame

-Incidence of AEs, including SAE -Change in growth parameters -Shifts from baseline in clinical laboratory parameters, ECGs, and vital signs -Shifts from baseline in coagulation parameters (aPTT, PT, and INR) -Change in urine total protein -Change from baseline in neurological examination outcomes -The proportion of participants with a postbaseline platelet count below the lower limit of normal on at least 2 consecutive measurements -The proportion of participants with a postbaseline QTcF of > 500 msec and an increase from baseline to any postbaseline timepoint in QTcF of > 60 msec

—

Secondary

Measure	Time frame
-Total number of new WHO motor milestones -Ventilator use -Time to death (overall survival) -Change from baseline in CHOP INTEND total score -Change from baseline in HINE Section 2 motor milestones -Proportion of HINE Section 2 motor milestone responders -Proportion of time on ventilation -Time to death or permanent ventilation (tracheostomyor >= 16 hours of ventilation/day continuously for >21 days in the absence of an acute reversible event) -Change from baseline in HFMSE score -Change from baseline in RULM score	—

Measure

Time frame

-Total number of new WHO motor milestones -Ventilator use -Time to death (overall survival) -Change from baseline in CHOP INTEND total score -Change from baseline in HINE Section 2 motor milestones -Proportion of HINE Section 2 motor milestone responders -Proportion of time on ventilation -Time to death or permanent ventilation (tracheostomyor >= 16 hours of ventilation/day continuously for >21 days in the absence of an acute reversible event) -Change from baseline in HFMSE score -Change from baseline in RULM score

—

Countries

Australia, Canada, Estonia, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Japan, Latvia, Lebanon, Poland, Russia, South Korea, Spain, Taiwan, Turkey, United Kingdom, United States

Contacts

Public ContactBiogen Japan Medical Information

Biogen Japan Ltd.

japan-medinfo@biogen.com+81-120-560-086

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026