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NCT07767890

A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients

StatusRecruiting
PhaseNot specified
Primary completion (estimated)
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Record History

All 3 versions: 3 with changes below.

  1. Recruitment opened
    • Trial sites expanded: 0 → 4 locations
    • Start date changed from estimate of Oct 30, 2026 to Oct 7, 2026
  2. Secondary endpoints: details revised at 6 of 7 entries
  3. Study First Submitted
  • Notable: the plan changed
  • Minor: routine updates

Endpoints

Primary

Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs)
Timeframe: Up to approximately 4 years and 5 months
Number of Patients With Serious Adverse Events and ADRs Corresponding to the Safety Specifications
Timeframe: Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs
Timeframe: Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs Corresponding to the Safety Specifications
Timeframe: Up to approximately 4 years and 5 months
Number of Patients by Time to Onset of Serious Adverse Events and ADRs
Timeframe: Up to approximately 4 years and 5 months
Number of Patients by Time to Onset of Serious Adverse Events and ADRs Corresponding to the Safety Specifications
Timeframe: Up to approximately 4 years and 5 months

Secondary (7)

Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score
Change From Baseline in Revised Upper Limb Module (RULM) Score
Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score
Change From Baseline in Clinical Global Impression-Severity (CGI-S) Score
Number of Patients Categorized by Clinical Global Impression-Improvement (CGI-I) Score
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Number of Patients Achieving Each Developmental Milestone
Number of Patients Maintaining Each Developmental Milestone

Arms and interventions

Onasemnogene Abeparvovec Intrathecal Group

SMA patients treated with onasemnogene abeparvovec intrathecal injection in Japan.

Sites

4 sites in 1 country