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New treatment option for Spinal Muscular Atrophy
Official title Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
ClinicalTrials.gov ID: NCT05866419
What this study is testing
- What it's testing
- 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.
- Time commitment: about 37 months
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 and older
You may be able to join 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...
- people for whom the treating physician determines implantation of the ThecaFlex DRx™ System is otherwise in the subject's best interest
You likely can't join 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...
- 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...
- Severe structural impediment that may preclude safe implantation of the catheter and port
- Major medical events within 60 days prior to screening; or
See the full eligibility criteria
Who can join
- 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
- people 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...
- 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.
What rules you out
- 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...
- 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
- 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
- History of hydrocephalus
- Diagnosed degenerative muscular disease other than SMA
- 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
- Serious medical condition that, in the opinion of the investigator, may lead to reduced life expectancy beyond 12 months.
- Subject is involved in another Investigation Device Exemption Study.
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- Orange, California, United States
- Palo Alto, California, United States
- San Diego, California, United States
- Orlando, Florida, United States
- Chicago, Illinois, United States
- Boston, Massachusetts, United States
- Grand Rapids, Michigan, United States
- New York, New York, United States
- Cleveland, Ohio, United States
- Hershey, Pennsylvania, United States
- Philadelphia, Pennsylvania, United States
- Houston, Texas, United States
- Charlottesville, Virginia, United States
- Norfolk, Virginia, United States
- Essen, Germany
- Krakow, Poland
- Lodz, Poland
- Madrid, Spain
- Valencia, Spain
+ 1 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 37 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; Orange, California, United States; Palo Alto, California, United States; San Diego, California, United States; Orlando, Florida, United States; Chicago, Illinois, United States and 15 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.