NCT05866419

Brief Summary

The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA). All enrolled subjects will undergo implantation of the investigational device (ThecaFlex DRx™ System) and will be followed for 12 months after receiving the implant. The 12-month data will be used to assess the primary endpoint support a Pre-Market Approval (PMA) application.

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
90

participants targeted

Target at P50-P75 for not_applicable

Timeline
50mo left

Started Nov 2023

Longer than P75 for not_applicable

Geographic Reach
5 countries

21 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress37%
Nov 2023Jul 2030

First Submitted

Initial submission to the registry

May 10, 2023

Completed
9 days until next milestone

First Posted

Study publicly available on registry

May 19, 2023

Completed
6 months until next milestone

Study Start

First participant enrolled

November 27, 2023

Completed
4.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2028

Expected
2.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2030

Last Updated

April 16, 2026

Status Verified

April 1, 2026

Enrollment Period

4.3 years

First QC Date

May 10, 2023

Last Update Submit

April 14, 2026

Conditions

Spinal Muscular AtrophySpine DeformityScoliosis

Outcome Measures

Primary Outcomes (2)

  • The proportion of subjects with successful Implantation of the ThecaFlex DRx™ Port and Catheter

    The primary outcome measures the proportion of subjects with successful implantation of the ThecaFlex DRx™ Port and Catheter System who are able to receive successful intrathecal therapy infusion(s).

    12 Months

  • The proportion of subjects with successful Intrathecal Therapy Infusion(s) through 12 months

    12 Months

Secondary Outcomes (5)

  • Proportion of subjects with reduced anesthesia and radiation exposure compared to repeat LP for nusinersen infusion in SMA subjects.

    12 months

  • Incidence of device-related adverse events (AEs)/complications through 12 months

    12 months

  • Incidence of procedural complications through 12 months

    12 months

  • Incidence of nusinersen-related AEs

    12 months

  • Mean duration of radiation exposure from implant through 12-month follow-up

    12 months

Study Arms (1)

ThecaFlex DRx Port and Catheter System

EXPERIMENTAL

Subjects who meet all of the inclusion and none of the exclusion would be eligible to receive the ThecaFlex DRx Port and Catheter System.

Device: ThecaFlex DRx System

Interventions

ThecaFlex DRx SystemDEVICE

The ThecaFlex DRx™ System consists of an implantable intrathecal catheter, and an implantable subcutaneous port, designed specifically to meet the implantation, Cerebrospinal Fluid (CSF) aspiration, and intrathecal delivery and dosing needs of patients (aged ≥ 3 years) requiring repeated/on-going intermittent chronic bolus intrathecal treatment for life threatening, debilitating Central Nervous System (CNS) disorders who are precluded from Lumbar Puncture (LP) due to complex spine conditions, or who are resistant to LP for therapy administration or CSF aspiration.

ThecaFlex DRx Port and Catheter System

Eligibility Criteria

Age3 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Subject is 3 years or older
  • Subject diagnosed with SMA who are candidates for Spinraza treatment as indicated in its label
  • Subject resistant to lumbar puncture (LP), where resistance is defined as:
  • Subject with respiratory issues or other comorbidities who is at an increased risk for complications due to the need for repeat anesthesia and imaging radiation exposure to safely perform LP; or
  • Subjects for whom the treating physician determines implantation of the ThecaFlex DRx™ System is otherwise in the subject's best interest
  • Subject has been prescribed nusinersen via chronic intrathecal bolus administration in accordance with the drug labeling and first port access of the ThecaFlex DRx system is planned within 2 weeks of implantation
  • Subject, per Investigator discretion, is able to undergo a percutaneous or open surgical procedure for index port and catheter implantation where at least 7 cm of the ThecaFlex DRx system catheter will be placed intrathecally
  • Subject (or subject's legally authorized representative) is willing and able to provide written informed consent; and
  • Subject and parent/caregiver able and willing to complete all clinical investigation procedures, measurements, and visits.

You may not qualify if:

  • Subject meets any of the contraindications for use of the ThecaFlex DRx™ System as outlined in the ThecaFlex DRx™ System Instructions for Use
  • Presence or history of (\< 6 months prior to the procedure): an implanted microinfusion pump, intrathecal catheter, or other intrathecal drug delivery devices/component, or implanted shunt for drainage of cerebrospinal fluid (CSF), or reservoir in the CSF space (note: any ThecaFlex Port and Catheter System must not be placed in the same location as a previous indwelling port and catheter)
  • Subject is pregnant or nursing or plans to become pregnant during the course of the clinical investigation
  • Any condition or event, which in the opinion of the Investigator may adversely affect the safety and effective implantation and use of the ThecaFlex DRx™ system or confound the clinical investigation, including:
  • Severe structural impediment that may preclude safe implantation of the catheter and port
  • Major medical events within 60 days prior to screening; or
  • Relevant surgeries (e.g., head and neck, back, etc.) within 60 days prior to screening or planned during the duration of the clinical investigation
  • Space-occupying lesion with mass effect
  • Posterior fossa mass
  • Arnold-Chiari malformation
  • Coagulation abnormalities and/or thrombocytopenia
  • Insufficient or inadequate skin or underlying fat to adequately protect the port or enough surface area to allow the skin to close safely
  • Subject is contraindicated for administration of nusinersen per its approved labeling
  • History of intrathecal granuloma formation
  • History of bacterial meningitis or aseptic meningitis within 6 months of screening
  • +6 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (21)

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States

RECRUITING

Children's Hospital Orange County

Orange, California, 92868, United States

RECRUITING

Stanford Medical Center

Palo Alto, California, 94301, United States

RECRUITING

Rady Children's Hospital

San Diego, California, 92037, United States

RECRUITING

Nemours Children's Hospital, Florida

Orlando, Florida, 32827, United States

RECRUITING

Lurie Childrens Hospital

Chicago, Illinois, 60611, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

RECRUITING

Columbia University Irving Medical Center/NewYork Presbyterian Hospital

New York, New York, 10032, United States

RECRUITING

UH Rainbow Babies and Children's Hospital

Cleveland, Ohio, 44106, United States

RECRUITING

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

RECRUITING

Children's Hospital Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Texas Children's Hospital

Houston, Texas, 77030, United States

RECRUITING

University of Virginia

Charlottesville, Virginia, 22903, United States

RECRUITING

Childrens Hospital of the King's Daughters

Norfolk, Virginia, 23510, United States

RECRUITING

Universitätsklinikum Essen

Essen, Germany

RECRUITING

Specialised Hospital Ludwika Rydygiera

Krakow, Poland

RECRUITING

Research Institute of Polish Mother's Memorial Hospital

Lodz, Poland

RECRUITING

Hospital Universitario La Paz

Madrid, Spain

RECRUITING

Hospital Universitario y Politecnico la Fe de Valencia

Valencia, 46026, Spain

RECRUITING

Sheffield Teaching Hospitals NHS Foundation Trust

Sheffield, United Kingdom

RECRUITING

Related Publications (1)

  • Mesina-Estarron I, Abruzzo M, Gomes FC, Thaxton M, Yengo-Kahn A, Stone S, Northam W, Snyder B, Cahill P, Bauer D, Muhonen M. Clinical evaluation of ThecaFlex DRx, a novel implantable catheter-port for intrathecal nusinersen delivery in spinal muscular atrophy: Initial results from the PIERRE-IDE study. J Neuromuscul Dis. 2026 Mar 13:22143602261432303. doi: 10.1177/22143602261432303. Online ahead of print.

    PMID: 41830093DERIVED

MeSH Terms

Conditions

Muscular Atrophy, SpinalScoliosis

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular DiseasesSpinal CurvaturesSpinal DiseasesBone DiseasesMusculoskeletal Diseases

Central Study Contacts

Janelle Arrambide

CONTACT

857-500-7526janelle.arrambide@biogen.com

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 10, 2023

First Posted

May 19, 2023

Study Start

November 27, 2023

Primary Completion (Estimated)

April 1, 2028

Study Completion (Estimated)

July 1, 2030

Last Updated

April 16, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

Locations

PL(2)DE(1)ES(2)US(15)GB(1)