Actively Recruiting
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)
Led by Biogen · Updated on 2026-03-18
58
Participants Needed
19
Research Sites
127 weeks
Total Duration
On this page
Sponsors
B
Biogen
Lead Sponsor
A
Alcyone Therapeutics, Inc
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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.
- Participant is 3 years old or older.
You will not qualify if you...
- Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) in other interventional clinical trials for SMA treatment (except the PIERRE study or myostatin inhibitor trials).
- 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.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 19 locations
1
Children's Hospital of Orange County
Orange, California, United States, 92868
Actively Recruiting
2
Stanford University Medical Center | Department of Neurology_Palo Alto
Palo Alto, California, United States, 94304
Actively Recruiting
3
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611-2605
Actively Recruiting
4
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
5
Helen DeVos Children's Hospita
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
6
Milton S. Hershey Medical Center | Pennsylvania State University_Hershey
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
7
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
8
Texas Childrens Hospital Houston
Houston, Texas, United States, 77030
Actively Recruiting
9
Stacey Hall Developmental Pediatrics
Charlottesville, Virginia, United States, 22903
Actively Recruiting
10
Children's Hospital of the King's Daughters_Norfolk
Norfolk, Virginia, United States, 23507
Actively Recruiting
11
Hôpital Raymond Poincaré
Garches, Hauts De Seine, France, 92380
Actively Recruiting
12
Universitaetsklinikum Essen
Essen, Germany, 45147
Actively Recruiting
13
Fondazione Serena Onlus - Centro Clinico Nemo_Milano
Milan, Italy, 20162
Withdrawn
14
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, Italy, 168
Withdrawn
15
Szpital Specjalistyczny im. L.Rydygiera w Krakowie
Krakow, Poland, 31-826
Not Yet Recruiting
16
Instytut Centrum Zdrowia Matki Polki
Lodz, Poland, 93-338
Not Yet Recruiting
17
Hospital Universitario La Paz
Madrid, Spain, 28046
Actively Recruiting
18
Hospital Universitari i Politecnic La Fe_Valencia
Valencia, Spain, 46026
Actively Recruiting
19
Royal Hallamshire Hospital Neurology Department
Sheffield, South Yorkshire, United Kingdom, S102JF
Actively Recruiting
Research Team
U
US Biogen Clinical Trial Center
CONTACT
G
Global Biogen Clinical Trial Center
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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