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

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

Iwasaki Ryohei

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

rinshoshiken.toroku@novartis.com

Iwasaki Ryohei

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

+81-120-003-293

rinshoshiken.toroku@novartis.com

Recruiting

Mar. 19, 2024

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

other

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.

There are no exclusion criteria for this study.

Both

Spinal Muscular Atrophy

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 includes Baseline through Year 5 visits, assessments will be performed at the Investigational site. During Follow-up Period 3 (Year 6 to up to Year 15 after OAV101 administration), participants/caregivers will be contacted using tele-visits annually for remote assessments. All patients will enter the study at the baseline visit and continue until 15 years since OAV101 administration is reached. Total duration of participation in the study will be dependent upon time of enrollment relative to OAV101 administration and will vary by participant.

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

+81-3-3353-8111

Feb. 09, 2024

Yes

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

Australia/Belgium