Memphis, TNNCT06532474Now EnrollingIRB Ready

Spinal Muscular Atrophy Clinical Trial in Memphis, TN

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

Sponsored by St. Jude Children's Research Hospital

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Expert Care in Memphis

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 Memphis, TN

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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 Memphis

    Convenient for TN 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 Memphis site if eligible
  4. 4Begin participation

About This Spinal Muscular Atrophy Study in Memphis

In this observational study, researchers are looking at the effects of spinal muscular atrophy (SMA) drugs on the muscles and nerve cells in patients with SMA. Primary Objectives * To evaluate the feasibility and reliability of performing MR functional imaging in exercising muscle in patients with SMA. * To evaluate patients with SMA types 2 and 3 at baseline and longitudinally at 6 and 12 months Secondary Objectives * To describe the MR functional bioenergetics response in muscles in five potential groups of patients with spinal muscular atrophy: untreated, actively treated with nusinersen (Spinraza®) or onasemnogene abeparvovec (Zolgensma®), actively treated with risdiplam (Evrysdi®), switching from Spinraza or Zolgensma to Evrysdi and initiating combination therapy of Spinraza or Zolgensma with Evrysdi . * To identify changes in motor function in patients with SMA types 2 and 3 who initiate treatment with risdiplam. * To obtain biomarkers in blood, urine, and muscle tissue to provide proof-of-concept support for risdiplam effect on skeletal muscle. * To obtain quality of life and disability data from participants in this study.

Sponsor: St. Jude Children's Research Hospital

Who Can Participate

Inclusion Criteria

Genetic confirmation of SMA with homozygous deletion of SMN1 or compound heterozygous deletion/mutation of SMN1
Two, three, or four copies of SMN2
Age 5 to 20 years
Non-ambulatory participants: maximum function sitting or standing with support, HFMSE score at screening between 10 and 45 points.
Ambulatory participants: minimum function of independent walking, able to walk unassisted a minimum of 100 meters at screening, HFMSE score at screening between 40 and 66.
SMN-directed therapy inclusion:
Current Evrysdi prescription (Group 1)
Must have Evrysdi prescription through their treating physician
If initiating combined therapy using Evrysdi with Spinraza or Zolgensma, must have not started Evrysdi treatment OR
Current Spinraza or Zolgensma prescription (Group 2)
For patients on Spinraza, must have been taking Spinraza for at least 12 months at screening (4 loading and 2 maintenance doses) and following the FDA-recommended dosing schedule
For patients on Zolgensma, must have been dosed at least one year prior to screening
Must have Spinraza or Zolgensma prescription through their treating physician OR
Changing from Spinraza or Zolgensma to Evrysdi (Group 3)
For patients on Spinraza, must have been taking Spinraza for at least 12 months at screening (4 loading and 2 maintenance doses) and following the FDA-recommended dosing schedule
For patients on Zolgensma, must have been dosed at least one year prior to screening
Must have voluntarily decided to switch therapies based on discussion with their treating physician
Must have Evrysdi prescription through their treating physician but have not yet initiated treatment OR
Have never received any SMN-directed therapies (Group 4)

Exclusion Criteria

Any chronic medical condition, planned surgery, or treatment with a medication which would impact safety or participation of the study at the investigator's discretion
Inability to perform reliably the motor function testing or the exercise testing in the MR scanner.
Fat fraction \> 35% in calf or bicep at screening MRI
Need for routine non-invasive ventilation support.
Non-oral nutritional support, e.g., gastrostomy tube feeding.
Any ferrous metal implants (e.g., spinal rods) that preclude testing in a MR scanner.

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 Memphis?

Yes, this clinical trial (NCT06532474) has an active research site in Memphis, TN 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 Memphis, TN

If you're searching for spinal muscular atrophy treatment options in Memphis, TN, this clinical trial (NCT06532474) 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 Memphis 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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