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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

Long-Term Extension Study of Nusinersen in Participants With SMA

Irzhevsky Victoria

Biogen Japan Ltd.

Nihonbashi 1-chome Mitsui Building 14F, 1-4-1, Nihonbashi, Chuo-ku, Tokyo, 103-0027

japan-medinfo@biogen.com

Biogen Japan Medical Information

Biogen Japan Ltd.

Nihonbashi 1-chome Mitsui Building 14F, 1-4-1, Nihonbashi, Chuo-ku, Tokyo, 103-0027

+81-120-560-086

japan-medinfo@biogen.com

Not Recruiting

May. 26, 2022

Interventional

non-randomized controlled trial

double blind

dose comparison control

parallel assignment

treatment purpose

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

- 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.

Both

Spinal muscular atrophy

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 Study 302. Those who received the currently approved maintenance dose of 12 mg nusinersen in Study 203 will be administered a bolus dose of 50 mg nusinersen on Day 1 followed by 28 mg nusinersen maintenance doses every 4 months thereafter, which is identical to the regimen in Part C of Study 203.

- 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

- 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

+81-3-5269-7839

Jan. 14, 2022

Yes

In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/

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