Long-Term Extension Study of Nusinersen in Participants With SMA

JRCT ID: jRCT2031220035

Registered date:23/04/2022

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Spinal muscular atrophy
• Date of first enrollment: 17/08/2022
• Target sample size: 172
• Countries of recruitment: United States,Japan,Canada,Japan,Estonia,Japan,Hungary,Japan,Ireland,Japan,Italy,Japan,Latvia,Japan,Poland,Japan,Spain,Japan,Taiwan,Japan,France,Japan,Germany,Japan,Greece,Japan,Australia,Japan,South Korea,Japan,United Kingdom,Japan,Israel,Japan,Lebanon,Japan,Russia,Japan,Turkey,Japan
• Study type: Interventional

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.

Outcome(s)

Primary Outcome

- 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

- 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

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: Not applicable
• Gender: Both
• Include criteria: Completion of the Day 302 Visit in Study 203 and compliance with the study protocol throughout Study 203.

Exclude 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.

Related Information

• Primary Sponsor: Irzhevsky Victoria
• Secondary ID(s): NCT04729907