Specified Use-Results Survey of Zolgensma Intrathecal Injection (Spinal Muscular Atrophy)
Trial Snapshot
- Phase
- Not Applicable
- Status
- Not yet recruiting
- Sponsor
- Novartis Pharmaceuticals
- Enrollment
- 80
- Primary Endpoint
- Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs)
Study Overview
Brief Summary
This study aims to assess the safety and effectiveness of onasemnogene abeparvovec intrathecal injection in spinal muscular atrophy (SMA) patients in clinical practice in Japan.
This is a non-interventional study and does not impose a therapy protocol, diagnostic/therapeutic procedure, or a visit schedule. Patients will be treated according to the Japan package insert. Data will be collected using a case registration form (CRF) completed by the investigator at each study site.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 2 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •All patients treated with onasemnogene abeparvovec.
Exclusion Criteria
- •Patients who have received onasemnogene abeparvovec for unapproved indications under the Clinical Trials Act or GCP (eg, investigator-initiated clinical trial).
Arms & Interventions
Onasemnogene Abeparvovec Intrathecal Group
SMA patients treated with onasemnogene abeparvovec intrathecal injection in Japan.
Outcomes
Primary Outcomes
Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs)
Time Frame: Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs
Time Frame: Up to approximately 4 years and 5 months
Incidence based on the person-year method will be calculated.
Incidence of Serious Adverse Events and ADRs Corresponding to the Safety Specifications
Time Frame: Up to approximately 4 years and 5 months
Incidence based on the person-year method will be calculated.
Number of Patients by Time to Onset of Serious Adverse Events and ADRs
Time Frame: 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
Time Frame: Up to approximately 4 years and 5 months
Number of Patients With Serious Adverse Events and ADRs Corresponding to the Safety Specifications
Time Frame: Up to approximately 4 years and 5 months
Safety specifications include hepatotoxicity, transient thrombocytopenia, thrombotic microangiopathy, cardiac adverse events, dorsal root ganglia toxicity/peripheral sensory neuropathy, tumorigenicity due to chromosomal integration, and long-term monitoring of gene therapy.
Secondary Outcomes
- Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score(Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)
- Change From Baseline in Revised Upper Limb Module (RULM) Score(Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)
- Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score(Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)
- Change From Baseline in Clinical Global Impression-Severity (CGI-S) Score(Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)
- Number of Patients Categorized by Clinical Global Impression-Improvement (CGI-I) Score(6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)
- Number of Patients Achieving Each Developmental Milestone(Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)
- Number of Patients Maintaining Each Developmental Milestone(Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)