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)
- Conditions
- Spinal Muscular Atrophy
- Registration Number
- 2024-514239-21-00
- Lead Sponsor
- Biogen Idec Research Limited
- Brief Summary
The main objective of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture. The main questions researchers want to answer are:
• What is the highest amount of nusinersen found in the blood after dosing?
• How much nusinersen is found in the blood over the first 24 hours after dosing?
- Detailed Description
The primary objective of this study is to assess the Pharmacokinetic (PK) profile of nusinersen delivered via standard LP and via the ThecaFlex DRx System in participants with SMA.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Not specified
- Target Recruitment
- 26
Participant is on regular maintenance dosing of nusinersen (12 milligrams [mg] dose) every 4 months, with 4 months (± 2 weeks) between the LP-delivered study dose and the last nusinersen dose prior to study enrollment.
Participants must be enrolled in the PIERRE study to be eligible for enrolment in the PIERRE PK study.
Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrolment in other interventional clinical trials for the treatment of SMA (except for the PIERRE study).
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 criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System
Area Under the Plasma Concentration-Time Curve From Zero Time to 24 Hours After Intrathecal Administration (AUC0-24h) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System Area Under the Plasma Concentration-Time Curve From Zero Time to 24 Hours After Intrathecal Administration (AUC0-24h) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (15)
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Stacey Hall Developmental Pediatrics
🇺🇸Charlottesville, Virginia, United States
Szpital Specjalistyczny im. L.Rydygiera w Krakowie
🇵🇱Krakow, Poland
Children's Hospital of Orange County
🇺🇸Orange, California, United States
Stanford University Medical Center | Department of Neurology_Palo Alto
🇺🇸Palo Alto, California, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Helen DeVos Children's Hospita
🇺🇸Grand Rapids, Michigan, United States
Milton S. Hershey Medical Center | Pennsylvania State University_Hershey
🇺🇸Hershey, Pennsylvania, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Texas Childrens Hospital Houston
🇺🇸Houston, Texas, United States
Scroll for more (5 remaining)Boston Children's Hospital🇺🇸Boston, Massachusetts, United StatesBasil T DarrasPrincipal Investigator