A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
This research study, called PIERRE-PK, is for people with Spinal Muscular Atrophy (SMA). We want to understand how a medicine called nusinersen moves through your body when it’s given using two different methods. One method is a standard spinal injection (lumbar puncture), and the other is a new device called the ThecaFlex DRx System. This device includes a small tube and a port placed under your skin. We'll compare how much nusinersen is in your blood and how long it stays there after each method. This study helps us learn more about how well new ways of giving medicine work for SMA. It's part of a larger ongoing study, PIERRE, that is already testing the ThecaFlex DRx system.
At a glance
What is this study about?
This study, called PIERRE-PK, is designed for people living with a condition called Spinal Muscular Atrophy (SMA). SMA affects the nerves that control muscles, leading to muscle weakness over time. There's a medicine called nusinersen that can help manage SMA, and it's usually given directly into the fluid surrounding the spinal cord through a procedure called a lumbar puncture (often shortened to LP).
Researchers are looking at a new way to give nusinersen using a device called the ThecaFlex DRx™ System. This system involves a tiny, flexible tube (catheter) that goes into the spinal fluid, connected to a small port that sits just under your skin. The idea is that once implanted, this system might make it easier to give the medicine without needing repeated lumbar punctures. This PIERRE-PK study wants to compare how effectively nusinersen travels through your body and reaches the right places when given by the standard lumbar puncture versus this new ThecaFlex DRx System.
By carefully checking how much nusinersen is in the blood after each method, the study aims to understand if the new system delivers the medicine in a similar way to the traditional method. This information is really important because it helps scientists learn if the ThecaFlex DRx System is a good option for people with SMA in the future. This study is linked to a larger study, called PIERRE, which is already testing the safety and overall use of the ThecaFlex DRx System.
Key takeaways
- This study compares two ways of giving SMA medicine (nusinersen): standard spinal injection vs. a new implantable system.
- It aims to understand how the medicine moves through the body with each method.
- Participation involves receiving nusinersen by both methods and providing blood samples.
- You must already be on stable nusinersen treatment and be part of the PIERRE study to join.
- The study lasts about 5 months and helps advance knowledge of SMA treatments.
- You can withdraw from the study at any time.
Who may be eligible?
To be able to join this study, you must already be taking nusinersen regularly for your SMA. Specifically, you need to be receiving the 12 milligram dose every four months, and four months (give or take a couple of weeks) should have passed since your last nusinersen dose before you start this study. It's also essential that you are already participating in the main PIERRE study to be considered for this PIERRE-PK part.
There are also some reasons why you wouldn't be able to join. For example, if you're currently in another study testing a different treatment for SMA (unless it's the PIERRE study or certain muscle-building medication studies), or if you've been in one within the last six months, you won't be eligible. You also can't join if you've never had nusinersen before, or if you're taking a different dose than 12 milligrams. If you've already had the ThecaFlex DRx system implanted, this particular study isn't for you. Lastly, if you are pregnant, breastfeeding, or planning to become pregnant during the study, you won't be able to take part.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you between 3 years old and any age for adults?
- Are you currently receiving a 12mg dose of nusinersen every 4 months?
- Has it been about 4 months since your last nusinersen dose (give or take 2 weeks)?
- Are you already participating in the main PIERRE study?
- Are you not pregnant, breastfeeding, or planning to become pregnant during the study?
- Have you not had the ThecaFlex DRx System implanted already?
What does participation involve?
If you decide to take part, the study will last about five months, and this time will happen alongside the first five months of your involvement in the main PIERRE study. First, you'll have an initial screening visit, which could take up to 30 days, to make sure you're a good fit for the study.
During the study, you'll receive a dose of nusinersen given with a standard spinal injection (lumbar puncture). After this, as part of the main PIERRE study, you'll have the ThecaFlex DRx System carefully put into your body. Then, at a later date, you'll receive another dose of nusinersen, this time given through your new ThecaFlex DRx System.
Throughout the study, both before and after each medicine dose, nurses or doctors will take small blood samples from you. The very last blood sample will be taken 24 hours after you've received a dose. These blood tests help the researchers understand how the medicine travels through your body.
Potential risks and benefits
Locations (19)
- Children's Hospital of Orange CountyVerified postcodeOrange, United States· Recruiting
- Stanford University Medical Center | Department of Neurology_Palo AltoVerified postcodePalo Alto, United States· Recruiting
- Ann & Robert H. Lurie Children's Hospital of ChicagoVerified postcodeChicago, United States· Recruiting
- Boston Children's HospitalVerified postcodeBoston, United States· Recruiting
- Helen DeVos Children's HospitaVerified postcodeGrand Rapids, United States· Recruiting
- Milton S. Hershey Medical Center | Pennsylvania State University_HersheyVerified postcodeHershey, United States· Recruiting
- Children's Hospital of PhiladelphiaVerified postcodePhiladelphia, United States· Recruiting
- Texas Childrens Hospital HoustonVerified postcodeHouston, United States· Recruiting
- Stacey Hall Developmental PediatricsVerified postcodeCharlottesville, United States· Recruiting
- Children's Hospital of the King's Daughters_NorfolkVerified postcodeNorfolk, United States· Recruiting
- Hôpital Raymond PoincaréVerified postcodeGarches, France· Recruiting
- Universitaetsklinikum EssenVerified postcodeEssen, Germany· Recruiting
Common questions
What is Spinal Muscular Atrophy (SMA)?
SMA is a genetic condition that causes muscle weakness and wasting because it affects nerve cells in the spinal cord that control movement.
What is nusinersen?
Nusinersen is a medicine approved to treat SMA. It works by helping the body produce a protein essential for healthy nerve function.
What is the ThecaFlex DRx System?
It's a new device being tested that includes a thin tube and a small port surgically placed under the skin to deliver medicines directly to the spinal fluid, potentially making regular treatments easier.
Why are blood samples needed?
Blood samples help researchers measure how much nusinersen is in your body and how it travels after it's been given in different ways.
How long will I be in this study?
The study will last around 5 months for each person, happening alongside the first part of your involvement in the larger PIERRE study.
How to find out more
US Biogen Clinical Trial Center
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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