San Diego, CANCT05866419Now EnrollingIRB Ready

Spinal Muscular Atrophy Clinical Trial in San Diego, CA

Access cutting-edge spinal muscular atrophy treatment through this clinical trial at a research site in San Diego. Study-provided care at no cost to qualified participants.

Sponsored by Alcyone Therapeutics, Inc

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Expert Care in San Diego

Access spinal muscular atrophy specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related spinal muscular atrophy treatment provided free

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Check if you qualify for this spinal muscular atrophy clinical trial in San Diego, CA

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Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to San Diego

    Convenient for CA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit San Diego site if eligible
  4. 4Begin participation

About This Spinal Muscular Atrophy Study in San Diego

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.

Sponsor: Alcyone Therapeutics, Inc

Who Can Participate

Inclusion Criteria

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.

Exclusion Criteria

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
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.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in San Diego?

Yes, this clinical trial (NCT05866419) has an active research site in San Diego, CA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Spinal Muscular Atrophy Treatment Options in San Diego, CA

If you're searching for spinal muscular atrophy treatment options in San Diego, CA, this clinical trial (NCT05866419) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our San Diego research site is actively enrolling participants for this clinical trial. You'll receive care from experienced spinal muscular atrophy specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all spinal muscular atrophy clinical trials near you to find additional studies recruiting in your area.

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