Long-Term Extension Study of Nusinersen in Participants With SMA

• Register: JPRN
• Last refreshed on: 17 October 2023
• Main ID: JPRN-jRCT2031220035
• Date of registration: 23/04/2022
• Prospective Registration: Yes
• Primary sponsor: Irzhevsky Victoria
• Public title: Long-Term Extension Study of Nusinersen in Participants With SMA
• Scientific title: 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
• Date of first enrolment: 17/08/2022
• Target sample size: 172
• Recruitment status: Not Recruiting
• URL: https://jrct.niph.go.jp/latest-detail/jRCT2031220035
• Study type: Interventional
• Study design: non-randomized controlled trial, double blind, dose comparison control, parallel assignment, treatment purpose
• Phase: 3

Countries of recruitment

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

• Name: Biogen Japan Medical Information
• Affiliation: Biogen Japan Ltd.

• Name: Victoria Irzhevsky
• Affiliation: Biogen Japan Ltd.

Key inclusion & exclusion criteria

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

Age minimum: Not applicable
Age maximum: Not applicable
Gender: Both

Health Condition(s) or Problem(s) studied

Spinal muscular atrophy

Intervention(s)

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.

Primary Outcome(s)

- 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 Outcome(s)

- 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

Secondary ID(s)

NCT04729907