Explore evaluation methods of Nusinersen efficacy on advanced SMA patients with limited gross motor function

Explore evaluation methods of Nusinersen efficacy on advanced SMA patients with limited gross motor function - NEOSMA Study

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-UMIN000033935

Enrollment

Registered

2018-08-29

Start date

2018-10-29

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

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1.Type-1 SMA patients who can provide written consent for participation in the study, whose ages are 3 years and older 2.Type-1 SMA patients who receive Nusinersen treatment or are determined to be subject to treatment 3.Type-1 SMA patients who are naive, and non-ambulatory, and Hammersmith Functional Motor Scale Expanded (HFMSE) score = 0 ,before receiving treatment

Exclusion criteria

Exclusion criteria: 1.Past medical history of Hypoxic brain injury or epileptic episode 2.Present respiratory infection 3.Patients who cannot perform matching test by eye gaze apparatus: unable to open eyes and to fix the eye gaze for the system

Design outcomes

Primary

Measure	Time frame
Matching pair test: Changes in scores (24 weeks after baseline) Changes in time taken for matching pair test (24 weeks after baseline)	—

Countries

Japan

Contacts

Public ContactSatoru Enomoto

Mebix, Inc. Research Promotion Group

neosma@mebix.co.jp03-4362-4504

Outcome results