Actively Recruiting
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
Led by Alcyone Therapeutics, Inc · Updated on 2026-04-16
90
Participants Needed
21
Research Sites
344 weeks
Total Duration
On this page
Sponsors
A
Alcyone Therapeutics, Inc
Lead Sponsor
B
Biogen
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subject is 3 years or older
- Diagnosed with spinal muscular atrophy (SMA) and a candidate for Spinraza treatment
- Resistant to lumbar puncture, defined as having respiratory or other health issues increasing risk with repeat anesthesia and imaging radiation, or deemed best for implantation by the physician
- Prescribed nusinersen for chronic intrathecal bolus administration with planned first port access within 2 weeks of implantation
- Able to undergo percutaneous or open surgical procedure with at least 7 cm of catheter placed intrathecally
- Willing and able to provide written informed consent
- Subject and parent/caregiver willing and able to complete all study procedures and visits
You will not qualify if you...
- Meets contraindications for use of the ThecaFlex DRx17 System as per Instructions for Use
- Has implanted microinfusion pump, intrathecal catheter, other intrathecal drug delivery devices, or CSF drainage shunt within 6 months prior to procedure
- Pregnant, nursing, or plans to become pregnant during the study
- Has severe structural issues precluding safe implantation, major medical events or relevant surgeries within 60 days prior to screening or planned during the study, space-occupying lesions, posterior fossa mass, Arnold-Chiari malformation, coagulation or platelet disorders, or inadequate skin/fat coverage for the port
- Contraindicated for nusinersen administration per labeling
- History of intrathecal granuloma formation
- History of bacterial or aseptic meningitis within 6 months of screening
- History of tumors or spinal abnormalities interfering with catheter implantation or CSF circulation
- History of hydrocephalus
- Diagnosed with degenerative muscular disease other than SMA
- History of depression, cognitive impairment, or other psycho-behavioral problems affecting safe participation
- Serious medical condition likely to reduce life expectancy beyond 12 months
- Currently involved in another Investigation Device Exemption Study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 21 locations
1
Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
Actively Recruiting
2
Children's Hospital Orange County
Orange, California, United States, 92868
Actively Recruiting
3
Stanford Medical Center
Palo Alto, California, United States, 94301
Actively Recruiting
4
Rady Children's Hospital
San Diego, California, United States, 92037
Actively Recruiting
5
Nemours Children's Hospital, Florida
Orlando, Florida, United States, 32827
Actively Recruiting
6
Lurie Childrens Hospital
Chicago, Illinois, United States, 60611
Actively Recruiting
7
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
8
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
9
Columbia University Irving Medical Center/NewYork Presbyterian Hospital
New York, New York, United States, 10032
Actively Recruiting
10
UH Rainbow Babies and Children's Hospital
Cleveland, Ohio, United States, 44106
Actively Recruiting
11
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
12
Children's Hospital Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
13
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
14
University of Virginia
Charlottesville, Virginia, United States, 22903
Actively Recruiting
15
Childrens Hospital of the King's Daughters
Norfolk, Virginia, United States, 23510
Actively Recruiting
16
Universitätsklinikum Essen
Essen, Germany
Actively Recruiting
17
Specialised Hospital Ludwika Rydygiera
Krakow, Poland
Actively Recruiting
18
Research Institute of Polish Mother's Memorial Hospital
Lodz, Poland
Actively Recruiting
19
Hospital Universitario La Paz
Madrid, Spain
Actively Recruiting
20
Hospital Universitario y Politecnico la Fe de Valencia
Valencia, Spain, 46026
Actively Recruiting
21
Sheffield Teaching Hospitals NHS Foundation Trust
Sheffield, United Kingdom
Actively Recruiting
Research Team
J
Janelle Arrambide
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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