A Study to Find Out How Nusinersen is Processed in the Bo... | NCT06555419

ID	Type	Description	Link
2024-514239-21-00	Other Identifier	EU CTIS Number

Status Module

Record Verification Date

Mar 2026

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 16, 2025Actual

Primary Completion Date

Jun 25, 2027Estimated

Completion Date

Jun 25, 2027Estimated

First Submitted Date

Aug 9, 2024

First Submission Date that Met QC Criteria

Aug 13, 2024

First Posted Date

Aug 15, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 16, 2026

Last Update Posted Date

Mar 18, 2026Actual

Name	Class
Alcyone Therapeutics, Inc	INDUSTRY

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

The primary objective of this study is to assess the Pharmacokinetic (PK) profile of nusinersen delivered via standard LP and via the ThecaFlex DRx System in participants with SMA.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

58Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Nusinersen Via LP and ThecaFlex DRx System	Experimental	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.	Drug: Nusinersen Device: ThecaFlex DRx System

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Nusinersen	Drug	Administered as specified in the treatment arm.	Nusinersen Via LP and ThecaFlex DRx System

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility 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 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.

NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

3 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
US Biogen Clinical Trial Center	Contact	866-633-4636		clinicaltrials@biogen.com
Global Biogen Clinical Trial Center	Contact			clinicaltrials@biogen.com

Overall Officials

Name	Affiliation	Role
Medical Director	Biogen	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Children's Hospital of Orange County	Recruiting	Orange	California	92868	United States

IPD Sharing Statement Module

Plan to Share IPD

Yes

Description

In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

https://vivli.org/

ID	Term
D013118	Spinal Cord Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D016472	Motor Neuron Disease
D019636	Neurodegenerative Diseases
D009468	Neuromuscular Diseases