An Open Label, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)

Status: Recruiting
Location: See all (18) locations...
Intervention Type: Drug, Device
Study Type: Interventional
Study Phase: Phase 1
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Healthy Volunteers: f
View:

• 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.

Locations
United States
California
Children's Hospital of Orange County
RECRUITING
Orange
Stanford University Medical Center | Department of Neurology_Palo Alto
RECRUITING
Palo Alto
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING
Chicago
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
Michigan
Helen DeVos Children's Hospita
RECRUITING
Grand Rapids
Pennsylvania
Milton S. Hershey Medical Center | Pennsylvania State University_Hershey
RECRUITING
Hershey
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Texas
Texas Childrens Hospital Houston
RECRUITING
Houston
Virginia
Stacey Hall Developmental Pediatrics
RECRUITING
Charlottesville
Children's Hospital of the King's Daughters_Norfolk
RECRUITING
Norfolk
Other Locations
France
Hôpital Raymond Poincaré
RECRUITING
Garches
Germany
Universitaetsklinikum Essen
RECRUITING
Essen
Italy
Fondazione Serena Onlus - Centro Clinico Nemo_Milano
RECRUITING
Milan
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
NOT_YET_RECRUITING
Roma
Poland
Szpital Specjalistyczny im. L.Rydygiera w Krakowie
RECRUITING
Krakow
Instytut Centrum Zdrowia Matki Polki
RECRUITING
Lodz
Spain
Hospital Universitario La Paz
RECRUITING
Madrid
Hospital Universitari i Politecnic La Fe_Valencia
RECRUITING
Valencia
Contact Information
Primary
US Biogen Clinical Trial Center
clinicaltrials@biogen.com
866-633-4636
Backup
Global Biogen Clinical Trial Center
clinicaltrials@biogen.com
Time Frame
Start Date: 2025-01-16
Estimated Completion Date: 2026-05-15
Participants
Target number of participants: 58
Treatments
Experimental: Nusinersen Via LP and ThecaFlex DRx System
Participants 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.
Sponsors
Collaborators: Alcyone Therapeutics, Inc
Leads: Biogen

This content was sourced from clinicaltrials.gov