臨床研究等提出・公開システム

A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam

A Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam

Tsuda Hiroshi

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

Pending

Jan. 04, 2024

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

- Genetic documentation of 5q SMA homozygous SMN1 gene deletion or mutation or compound heterozygous mutation.
- Diagnosis of later-onset SMA with symptom onset at age > 6 months.
- Aged >= 15 to <= 50 years at the time of informed consent.
- Body weight > 20 kg.
- Received oral risdiplam per the approved label or per the managed access program as follows:
- Nusinersen-naive participants must have had prior treatment with risdiplam for >= 6 months before enrollment.
- Nusinersen-experienced participants must have stopped nusinersen for >= 16 months and must have been on risdiplam for >= 12 months before enrollment.
- Able to perform the age-appropriate functional assessments in the study.
- RULM entry item A score >= 3.
- RULM total score >= 5 and <= 30 at Screening.
- Nonambulatory, defined as not able to walk 15 feet (4.57 meters) independently without support.
- Willing to stop risdiplam treatment.
- Willing and able to start treatment with HD nusinersen.

NOTE: Other protocol defined Inclusion criteria may apply.

- Any major illness within 1 month before the screening examination or within 1 week prior to Screening and up to first dose administration.
- Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the Screening Period.
- Presence of an implanted shunt for the drainage of CSF or of an implanted central nervous system catheter.
- Permanent tracheostomy or permanent ventilation at Screening.
- The medical necessity, as defined by the Investigator, for noninvasive ventilation such as bilevel positive airway pressure or continuous positive airway pressure outside of regular sleep hours for any reason other than proactive SMA management, at Screening.
- History of bacterial meningitis, viral encephalitis, or hydrocephalus.
- Ongoing medical condition that according to the Investigator would interfere with the conduct and assessments of the study. An example is a medical disability (e.g., wasting or cachexia, severe anemia, and respiratory parameters) that would interfere with the assessment of safety or would compromise the ability of the participant to undergo study procedures.

Both

Spinal muscular atrophy (SMA)

Research Name: BIIB058
Generic Name: Nusinersen
Trade Name: Spinraza

During the study, 2 loading doses of 50 mg nusinersen will be administered approximately 2 weeks apart followed by maintenance doses of 28 mg nusinersen administered approximately 4 months.

- Change in total RULM score

- Incidence of AEs, including SAEs
- Change in clinical laboratory parameters, ECGs, vital signs from baseline

+81-6-6853-2001

July. 21, 2023

Yes

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

USA/Belgium/Italy/Germany/Poland/Spain/Brazil/Hungary