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)

Part of paid clinical trials in Orange, California.

Sponsor
Biogen
Study ID
NCT06555419
Phase
PHASE1
Status
Recruiting
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Conditions

  • Muscular Atrophy, Spinal

Eligibility Criteria

Sex
ALL
Age
3 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Nusinersen — DRUG
    Administered as specified in the treatment arm.
  • ThecaFlex DRx System — DEVICE
    Implanted as specified in the treatment arm.

Study Details

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.

Key Dates

Start date
Jan 16, 2025
Status verified
Mar 2026
Primary completion
Jun 25, 2027
Completion
Jun 25, 2027

Study Design

Enrollment
58 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • 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.

Primary Outcome Measure

Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System [ Time Frame: Pre-dose and at multiple time points post-dose up to 4 months ]

Central Contacts

Locations (10)

FacilityCityStateZIPSite coordinators
Children's Hospital of Orange CountyOrangeCalifornia92868
714-509-3605
Julian Thomas (PRINCIPAL_INVESTIGATOR)
Stanford University Medical Center | Department of Neurology_Palo AltoPalo AltoCalifornia94304
650-723-5152
John Day (PRINCIPAL_INVESTIGATOR)
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611-2605
312-227-3550
Nancy Kuntz (PRINCIPAL_INVESTIGATOR)
Boston Children's HospitalBostonMassachusetts02115
617-355-8036
Basil T Darras (PRINCIPAL_INVESTIGATOR)
Helen DeVos Children's HospitaGrand RapidsMichigan49503
616-391-2241
Jena Krueger (PRINCIPAL_INVESTIGATOR)
Milton S. Hershey Medical Center | Pennsylvania State University_HersheyHersheyPennsylvania17033
717-232-5443
Dustin Paul (PRINCIPAL_INVESTIGATOR)
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
215-590-3174
Patrick Cahill (PRINCIPAL_INVESTIGATOR)
Texas Childrens Hospital HoustonHoustonTexas77030
832-822-1750
David Bauer (PRINCIPAL_INVESTIGATOR)
Stacey Hall Developmental PediatricsCharlottesvilleVirginia22903
434-924-8184
Rebecca J Scharf (PRINCIPAL_INVESTIGATOR)
Children's Hospital of the King's Daughters_NorfolkNorfolkVirginia23507
757-668-7000
Crystal Proud (PRINCIPAL_INVESTIGATOR)

Find similar trials in Orange, CA

By research site
Children's Hospital of Orange County· Orange, CAStanford University Medical Center | Department of Neurology_Palo Alto· Palo Alto, CAAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILBoston Children's Hospital· Boston, MAHelen DeVos Children's Hospita· Grand Rapids, MIMilton S. Hershey Medical Center | Pennsylvania State University_Hershey· Hershey, PA

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