Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen (ONWARD)

Biogen

Status and phase: Invitation-only; Phase 3

Conditions: Muscular Atrophy, Spinal

Treatments: Drug: Nusinersen

Study type: Interventional

Funder types: Industry

Identifiers

NCT04729907

2023-505637-27 (Other Identifier)

232SM302

Details and patient eligibility

About

The primary objective of this study is to evaluate the long-term safety and tolerability of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA) who previously participated in study 232SM203 (NCT04089566).

The secondary objective of this study is to evaluate the long-term efficacy of nusinersen administered intrathecally at higher doses to participants with SMA who previously participated in study 232SM203 (NCT04089566).

Enrollment: 145 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Completed the Day 302 visit in study 232SM203 (NCT04089566) in accordance with the study protocol

Key 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 232SM203 (NCT04089566)

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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145 participants in 2 patient groups

BIIB058 28 mg (Prior Maintenance Dose 28 mg)

Experimental group

Description:

Participants who received maintenance dose of 28 milligrams (mg) nusinersen in study 232SM203 (NCT04089566), will receive maintenance dose of 28 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 1921.

Treatment:

Drug: Nusinersen

BIIB058 50/28 mg (Prior Maintenance Dose 12 mg)

Experimental group

Description:

Participants who received maintenance dose of 12 mg nusinersen in study 232SM203 (NCT04089566), will receive loading dose of 50 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 1921.

Treatment:

Drug: Nusinersen