Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy

This study is looking for people aged 3 years or older who have Spinal Muscular Atrophy (SMA) and are already receiving or are about to start treatment with nusinersen. A key requirement is that you find it difficult to have your usual spinal injections, maybe because of breathing problems, other health conditions, or if your doctor believes this new device would be better for you.

You also need to be well enough to have a small operation to put the device in. You or your legal guardian must be willing to understand and sign consent forms, and be able to attend all clinic visits and complete study procedures.

Things that might stop you from joining include being pregnant or breastfeeding, or having certain other medical devices already implanted around your spine. Your doctor will carefully review your health to make sure this study is right for you.

If you join this study, you'll first go through a screening period, which could last up to about 6 and a half months, to make sure you're a good fit. After that, if you're eligible, you'll have a small operation to implant the ThecaFlex DRx System. This device will stay in for the duration of the study.

Once the device is in, you will receive your nusinersen medicine through it. If you've been taking nusinersen before, you'll likely receive your maintenance doses through the device every four months. If you're new to nusinersen, you'll receive your first few doses more frequently (three doses 14 days apart, then a fourth dose 30 days later) and then maintenance doses every four months.

You'll be carefully monitored for 12 months after the device is implanted, with regular visits to the clinic. Over this year, independent groups of experts will also review the safety information collected from everyone in the study.