Study of Nusinersen Among Patients with Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec

A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec

Status

Active, not recruiting

Phases

Phase 4

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2051210186

Enrollment

Registered

2022-03-03

Start date

2022-03-31

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 (SMA)

Interventions

Research Name: BIIB058 Generic Name: Nusinersen Trade Name: Spinraza Participants will undergo a loading period during which they will receive 12 mg nusinersen administered IT on Days 1,15, 29, and 64

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Genetic documentation of 5q SMA homozygous SMN1 gene deletion or mutation or compound heterozygous mutation. - Must have received onasemnogene abeparvovec. - Suboptimal clinical status as determined by the Investigator.

Exclusion criteria

Exclusion criteria: - Severe or serious AEs related to onasemnogene abeparvovec therapy that were ongoing. - Prior exposure to nusinersen.

Design outcomes

Primary

Measure	Time frame
- Total HINE Section 2 motor milestones score Section 2 of the HINE is composed of 8 motor milestone categories: voluntary grasp, ability to kick in supine position, head control, rolling, sitting, crawling, standing, and walking.	—

Secondary

Measure	Time frame
- Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs). An AE is any untoward medical occurrence in a patient or clinical investigation subject (participant) administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal assessment such as an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose: - Results in death - In the view of the Investigator, places the participant at immediate risk of death. - Requires inpatient hospitalization or prolongation of existing hospitalization - Results in persistent or significant disability/incapacity - Results in a birth defect - Is a medically important event - Change from baseline in clinical laboratory parameters, ECGs, and vital sign - WHO motor milestones - Change from baseline in applicable motor function assessments: CHOP INTEND, HFMSE, and RULM The CHOP INTEND test was designed to evaluate the motor skills of infants with significant motor weakness, including infants with SMA. The CHOP INTEND test captures neck, trunk, and proximal and distal limb strength in 14 elicited and 2 observational items. The HFMSE is a reliable and validated tool used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with Type II and Type III SMA with limited ambulation to give objective information on motor ability and clinical progression. The RULM is an outcome measure developed to assess upper limb functional abilities in patients with SMA. This test consists of upper limb performance items that are reflective of activities of daily living. - Time to death or permanent ventilation (trache	—

Measure

Time frame

- Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs). An AE is any untoward medical occurrence in a patient or clinical investigation subject (participant) administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal assessment such as an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose: - Results in death - In the view of the Investigator, places the participant at immediate risk of death. - Requires inpatient hospitalization or prolongation of existing hospitalization - Results in persistent or significant disability/incapacity - Results in a birth defect - Is a medically important event - Change from baseline in clinical laboratory parameters, ECGs, and vital sign - WHO motor milestones - Change from baseline in applicable motor function assessments: CHOP INTEND, HFMSE, and RULM The CHOP INTEND test was designed to evaluate the motor skills of infants with significant motor weakness, including infants with SMA. The CHOP INTEND test captures neck, trunk, and proximal and distal limb strength in 14 elicited and 2 observational items. The HFMSE is a reliable and validated tool used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with Type II and Type III SMA with limited ambulation to give objective information on motor ability and clinical progression. The RULM is an outcome measure developed to assess upper limb functional abilities in patients with SMA. This test consists of upper limb performance items that are reflective of activities of daily living. - Time to death or permanent ventilation (trache

—

Countries

Germany, Israel, Italy, Japan, Spain, 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