Long-term Follow-up of Patients with Spinal Muscular Atrophy Treated with OAV101 in Clinical trials

Long-term Follow-up of Patients with Spinal Muscular Atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2033230642

Enrollment

Registered

2024-02-16

Start date

2024-03-19

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

Interventions

The study is comprised of a Baseline Period and 3 Follow-up Periods. Follow-up Periods 1 and 2 consist of in-person visits and Period 3 consists of tele-visits. Follow-up Periods 1 and 2, which includ

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Participated in an OAV101 clinical trial. 2. Written informed consent must be obtained before any assessment is performed. 3. Patient/Parent/legal guardian willing and able to comply with study procedures.

Exclusion criteria

Exclusion criteria: There are no exclusion criteria for this study.

Design outcomes

Primary

Measure	Time frame
Number of participants with treatment-emergent serious adverse events (SAEs) [Time Frame: Up to 15 years]	—

Countries

Australia, Belgium, Japan

Contacts

Public ContactRyohei Iwasaki

Novartis Pharma. K.K.

rinshoshiken.toroku@novartis.com+81-120-003-293

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026