BridgeMD
New treatment option for Muscular Atrophy, Spinal
Official title 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)
ClinicalTrials.gov ID: NCT06555419
What this study is testing
What is Nusinersen?
Nusinersen is an investigational medicine, being studied as a potential treatment for muscular atrophy, spinal.
Also referred to as BIIB058.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP). The ThecaFlex DRx system is an investigational implantable medical device developed by Alcyone Therapeutics, Inc.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 and older
You may be able to join if
- Participant is on regular maintenance dosing of nusinersen (12 milligrams [mg] dose) every 4 months, with 4 months (± 2 weeks) between the...
- Participants must be enrolled in the PIERRE study to be eligible for enrolment in the PIERRE PK study. Key
You likely can't join if
- Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other treatment clinical...
- Participant is naïve to nusinersen treatment.
- Participant is receiving nusinersen at a dose other than 12 mg.
- Participant has already undergone implantation of the ThecaFlex DRx system.
- Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study. NOTE: Other protocol-defined Inclusion/Exclusion...
The study team makes the final eligibility decision.
Where it's taking place
- Orange, California, United States
- Palo Alto, California, United States
- Chicago, Illinois, United States
- Boston, Massachusetts, United States
- Grand Rapids, Michigan, United States
- Hershey, Pennsylvania, United States
- Philadelphia, Pennsylvania, United States
- Houston, Texas, United States
- Charlottesville, Virginia, United States
- Norfolk, Virginia, United States
- Garches, Hauts De Seine, France
- Essen, Germany
- Milan, Italy
- Roma, Italy
- Krakow, Poland
- Lodz, Poland
- Madrid, Spain
- Valencia, Spain
- Sheffield, South Yorkshire, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Orange, California, United States; Palo Alto, California, United States; Chicago, Illinois, United States; Boston, Massachusetts, United States; Grand Rapids, Michigan, United States; Hershey, Pennsylvania, United States and 13 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.