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)
PIERRE-PK
An Open Label, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)
2 other identifiers
interventional
58
7 countries
19
Brief Summary
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. It consists of a catheter, which is a flexible tube, connected to a port which is placed under the skin. Alcyone Therapeutics, Inc. has an ongoing study called PIERRE to test the ThecaFlex DRx system. Participants with spinal muscular atrophy (SMA) in the PIERRE study may be enrolled in the PIERRE-PK study. 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? The PIERRE-PK study will be done as follows:
- Participants will be screened to check if they can join the study. The screening period will be up to 30 days for this study and may overlap with the PIERRE study.
- Participants will receive a dose of nusinersen by lumbar puncture.
- The ThecaFlex DRx system will be implanted after the lumbar puncture, as part of the PIERRE study.
- Participants will receive a dose of nusinersen by the ThecaFlex DRx system, as part of the PIERRE study.
- Researchers will take blood samples before and after each dose. The last blood sample will be taken 24 hours after the dose.
- The total study duration for each participant in the PIERRE-PK study will be approximately 5 months. This period will overlap with the participant's first 5 months in the PIERRE study.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Jan 2025
Typical duration for phase_1
19 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 9, 2024
CompletedFirst Posted
Study publicly available on registry
August 15, 2024
CompletedStudy Start
First participant enrolled
January 16, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 25, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 25, 2027
March 18, 2026
March 1, 2026
2.4 years
August 9, 2024
March 16, 2026
Conditions
Outcome Measures
Primary Outcomes (2)
Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System
Pre-dose and at multiple time points post-dose up to 4 months
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
Pre-dose and at multiple time points post-dose up to 4 months
Study Arms (1)
Nusinersen Via LP and ThecaFlex DRx System
EXPERIMENTALParticipants will receive a maintenance dose of nusinersen, 12 milligrams (mg) via LP in the PIERRE-PK study, followed by implantation of the ThecaFlex DRx System and the subsequent nusinersen 12 mg maintenance dose via the system in the PIERRE study.
Interventions
Administered as specified in the treatment arm.
Implanted as specified in the treatment arm.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other interventional clinical trials for the treatment of SMA (except for the PIERRE study or interventional clinical trials of myostatin inhibitors \[e.g., apitegromab and taldefgrobep alfa\]).
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Biogenlead
- Alcyone Therapeutics, Inccollaborator
Study Sites (19)
Children's Hospital of Orange County
Orange, California, 92868, United States
Stanford University Medical Center | Department of Neurology_Palo Alto
Palo Alto, California, 94304, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611-2605, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Helen DeVos Children's Hospita
Grand Rapids, Michigan, 49503, United States
Milton S. Hershey Medical Center | Pennsylvania State University_Hershey
Hershey, Pennsylvania, 17033, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Texas Childrens Hospital Houston
Houston, Texas, 77030, United States
Stacey Hall Developmental Pediatrics
Charlottesville, Virginia, 22903, United States
Children's Hospital of the King's Daughters_Norfolk
Norfolk, Virginia, 23507, United States
Hôpital Raymond Poincaré
Garches, Hauts De Seine, 92380, France
Universitaetsklinikum Essen
Essen, 45147, Germany
Fondazione Serena Onlus - Centro Clinico Nemo_Milano
Milan, 20162, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 168, Italy
Szpital Specjalistyczny im. L.Rydygiera w Krakowie
Krakow, 31-826, Poland
Instytut Centrum Zdrowia Matki Polki
Lodz, 93-338, Poland
Hospital Universitario La Paz
Madrid, 28046, Spain
Hospital Universitari i Politecnic La Fe_Valencia
Valencia, 46026, Spain
Royal Hallamshire Hospital Neurology Department
Sheffield, South Yorkshire, S102JF, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director
Biogen
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 9, 2024
First Posted
August 15, 2024
Study Start
January 16, 2025
Primary Completion (Estimated)
June 25, 2027
Study Completion (Estimated)
June 25, 2027
Last Updated
March 18, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will share
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/