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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)

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Biogen

Status and phase

Begins enrollment in 1 month
Phase 1

Conditions

Muscular Atrophy, Spinal

Treatments

Drug: Nusinersen
Device: ThecaFlex DRx System

Study type

Interventional

Funder types

Industry

Identifiers

NCT06555419
2024-514239-21-00 (Other Identifier)
295SM101

Details and patient eligibility

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 up to 5 months. This period will overlap with the participant's first 5 months in the PIERRE study.

Enrollment

55 estimated patients

Sex

All

Ages

3+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

  • 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

Key Exclusion Criteria:

  • 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

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

55 participants in 1 patient group

Nusinersen Via LP and ThecaFlex DRx System
Experimental group
Description:
Participants will receive a nusinersen maintenance dose via LP in the PIERRE-PK study, followed by implantation of the ThecaFlex DRx System and the subsequent nusinersen maintenance dose via the system in the PIERRE study.
Treatment:
Device: ThecaFlex DRx System
Drug: Nusinersen

Trial contacts and locations

0

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Central trial contact

US Biogen Clinical Trial Center; Global Biogen Clinical Trial Center

Data sourced from clinicaltrials.gov

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