NCT07767890

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.

Trial Health

65
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
80

participants targeted

Target at P50-P75 for all trials

Timeline
54mo left

Started Oct 2026

Longer than P75 for all trials

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 11, 2026

Completed
6 days until next milestone

First Posted

Study publicly available on registry

August 17, 2026

Completed
2 months until next milestone

Study Start

First participant enrolled

October 30, 2026

Expected
4.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 31, 2031

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 31, 2031

Last Updated

September 17, 2026

Status Verified

September 1, 2026

Enrollment Period

4.4 years

First QC Date

August 11, 2026

Last Update Submit

September 14, 2026

Conditions

Spinal Muscular Atrophy

Keywords

Onasemnogene Abeparvovec

Outcome Measures

Primary Outcomes (6)

  • Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs)

    Up to approximately 4 years and 5 months

  • Number of Patients With Serious Adverse Events and ADRs Corresponding to the Safety Specifications

    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.

    Up to approximately 4 years and 5 months

  • Incidence of Serious Adverse Events and ADRs

    Incidence based on the person-year method will be calculated.

    Up to approximately 4 years and 5 months

  • Incidence of Serious Adverse Events and ADRs Corresponding to the Safety Specifications

    Incidence based on the person-year method will be calculated.

    Up to approximately 4 years and 5 months

  • Number of Patients by Time to Onset of Serious Adverse Events and ADRs

    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

    Up to approximately 4 years and 5 months

Secondary Outcomes (7)

  • 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

  • +2 more secondary outcomes

Study Arms (1)

Onasemnogene Abeparvovec Intrathecal Group

SMA patients treated with onasemnogene abeparvovec intrathecal injection in Japan.

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

SMA patients administered onasemnogene abeparvovec by intrathecal injection in Japan.

You may qualify if:

  • All patients treated with onasemnogene abeparvovec.

You may not qualify if:

  • Patients who have received onasemnogene abeparvovec for unapproved indications under the Clinical Trials Act or GCP (eg, investigator-initiated clinical trial).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 11, 2026

First Posted

August 17, 2026

Study Start (Estimated)

October 30, 2026

Primary Completion (Estimated)

March 31, 2031

Study Completion (Estimated)

March 31, 2031

Last Updated

September 17, 2026

Record last verified: 2026-09

Data Sharing

IPD Sharing
Will not share