Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies

Recruiting Observational Study
Spinal Muscular Atrophy
No Placebo Group Every participant receives an active treatment — no one gets a placebo. No Study Drug Researchers observe your health over time — no experimental treatment is given.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
5 – 20
Sex
Any
Study type
Observational
Participants needed
24 (estimated)
Sponsor
St. Jude Children's Research Hospital · Other
Think this trial could be right for you?

Answer a few quick questions to see if you may meet the eligibility requirements.

Check Your Eligibility
About This Trial
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 month…
Trial Locations
Loading…

Loading trial locations…

Eligibility Criteria
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 b…
Contacts

Jean Laboe, RN

901-595-1693

referralinfo@stjude.org

CONTACT