Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
NCT ID: NCT05866419
Last Updated: 2025-12-26
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
NA
90 participants
INTERVENTIONAL
2023-11-27
2027-03-31
Brief Summary
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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.
Detailed Description
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The subject population will include subjects aged ≥ 3 years with SMA who are considered candidates for intrathecal port and catheter implantation because they require chronic, bolus intrathecal administration of necessary therapy, and who meet all the inclusion/exclusion criteria for the clinical investigation.
After a screening period of up to 200 days, all enrolled subjects will undergo implantation of the investigational device (ThecaFlex DRx™ System) and will be followed for 12 months after receiving the implant.
In agreement with the nusinersen dosing information, the treatment frequency will be adapted for non-naïve and naïve subjects:
Subjects who are not naïve to nusinersen should have nusinersen maintenance doses administered via the implant every 4 months (i.e., 4, 8, and 12 months after their last nusinersen dose, before enrollment in the clinical investigation).
Subjects who are naïve to nusinersen will have their first three loading doses administered via the implant at 14-day intervals. The fourth loading dose should be administered 30 days after the third dose. A maintenance dose should be administered once every 4 months thereafter within the 12 months post-implantation.
A DMC and Clinical Events Committee (CEC), independent of each other and the Sponsor, will be used to perform ongoing consistent adjudication of events related to the safety throughout the entire duration of the clinical investigation and to periodically review data that relate to the safety of the study.
The clinical investigation will enroll 90 subjects. The clinical investigation incorporates a lead-in phase which consists of 10 subjects enrolled, implanted, and followed for 30-days. Enrollment for the lead-in phase is expected to take approximately 6 to 9 months, at which point the Data Monitoring Committee (DMC) will review the data for safety and approve continuation of enrollment. The second phase of enrollment is expected to take approximately 12 to 15 months.
Individual subjects are anticipated to be enrolled in the clinical investigation for approximately 37 months. The total duration of this clinical investigation is estimated to be approximately 60 months.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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ThecaFlex DRx Port and Catheter System
Subjects who meet all of the inclusion and none of the exclusion would be eligible to receive the ThecaFlex DRx Port and Catheter System.
ThecaFlex DRx System
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.
Interventions
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ThecaFlex DRx System
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.
Eligibility Criteria
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Inclusion Criteria
2. Subject diagnosed with SMA who are candidates for Spinraza treatment as indicated in its label
3. Subject resistant to lumbar puncture (LP), where resistance is defined as:
1. 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
2. Subjects for whom the treating physician determines implantation of the ThecaFlex DRx™ System is otherwise in the subject's best interest
4. 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
5. 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
6. Subject (or subject's legally authorized representative) is willing and able to provide written informed consent; and
7. Subject and parent/caregiver able and willing to complete all clinical investigation procedures, measurements, and visits.
Exclusion Criteria
2. 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)
3. Subject is pregnant or nursing or plans to become pregnant during the course of the clinical investigation
4. 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:
1. Severe structural impediment that may preclude safe implantation of the catheter and port
2. Major medical events within 60 days prior to screening; or
3. Relevant surgeries (e.g., head and neck, back, etc.) within 60 days prior to screening or planned during the duration of the clinical investigation
4. Space-occupying lesion with mass effect
5. Posterior fossa mass
6. Arnold-Chiari malformation
7. Coagulation abnormalities and/or thrombocytopenia
8. Insufficient or inadequate skin or underlying fat to adequately protect the port or enough surface area to allow the skin to close safely
5. Subject is contraindicated for administration of nusinersen per its approved labeling
6. History of intrathecal granuloma formation
7. History of bacterial meningitis or aseptic meningitis within 6 months of screening
8. History of tumors or other spinal abnormalities documented by magnetic resonance imaging (MRI) or computed tomography (CT) that would interfere with the catheter implantation procedures or CSF circulation
9. History of hydrocephalus
10. Diagnosed degenerative muscular disease other than SMA
11. History of depression, cognitive impairment, or another psycho-behavioral problem that in the opinion of the Investigator may preclude safe participation in the clinical investigation and
12. Serious medical condition that, in the opinion of the investigator, may lead to reduced life expectancy beyond 12 months.
13. Subject is involved in another Investigation Device Exemption Study.
3 Years
ALL
No
Sponsors
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Biogen
INDUSTRY
Alcyone Therapeutics, Inc
INDUSTRY
Responsible Party
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Locations
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Barrow Neurological Institute
Phoenix, Arizona, United States
Children's Hospital Orange County
Orange, California, United States
Stanford Medical Center
Palo Alto, California, United States
Rady Children's Hospital
San Diego, California, United States
Lurie Childrens Hospital
Chicago, Illinois, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Columbia University Irving Medical Center/NewYork Presbyterian Hospital
New York, New York, United States
UH Rainbow Babies and Children's Hospital
Cleveland, Ohio, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Children's Hospital Philadelphia
Philadelphia, Pennsylvania, United States
Texas Children's Hospital
Houston, Texas, United States
University of Virginia
Charlottesville, Virginia, United States
Childrens Hospital of the King's Daughters
Norfolk, Virginia, United States
Universitätsklinikum Essen
Essen, , Germany
Centro Clinico Nemo
Milan, , Italy
Specialised Hospital Ludwika Rydygiera
Krakow, , Poland
Research Institute of Polish Mother's Memorial Hospital
Lodz, , Poland
Hospital Universitario La Paz
Madrid, , Spain
Hospital Universitario y Politecnico la Fe de Valencia
Valencia, , Spain
Countries
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Central Contacts
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Facility Contacts
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Kathleen Nagaran Hildebrand
Role: primary
Charlotte Lynskey
Role: primary
Habib Mofakham fini
Role: primary
Mariah Stechschulte
Role: primary
Raven HIll
Role: primary
Role: backup
Adam Porter
Role: primary
Natasha Schafer
Role: primary
Kimbery Berry
Role: primary
Abigail Schlosser
Role: primary
Brennan Harding
Role: primary
Samantha Gogel
Role: primary
Sarah Wisor
Role: primary
Chelsea Masterson
Role: primary
Role: backup
Melinda Griggs
Role: primary
Jaqueline Lipka
Role: primary
Michela Nani
Role: primary
Ryszard Nowak
Role: primary
Lukasz Przyslo
Role: primary
María del Mar García Romero
Role: primary
Nancy Ñungo Garzon
Role: primary
Other Identifiers
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QT-0179
Identifier Type: -
Identifier Source: org_study_id