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NCT06532474 · ClinicalTrials.gov registry record
Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
A clinical trial of Spinal Muscular Atrophy, sponsored by St. Jude Children's Research Hospital.
- Recruiting
- Registry status
- 24
- Enrollment target
- 1
- Study location
NCT06532474 is a study of Spinal Muscular Atrophy that is actively recruiting participants, run by St. Jude Children's Research Hospital. The registered enrollment target is 24 participants, below the 108-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (78% lower). The trial reports 1 study location across 1 state.
The verdict
NCT06532474, a study of Spinal Muscular Atrophy, is actively recruiting participants, sponsored by St. Jude Children's Research Hospital.
- RECRUITING
- Registry status
- 24 participants
- Enrollment target
- 1
- Study location
Study Summary
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 the leg muscles in four potential groups of patients with spinal muscular atrophy: untreated, actively treated with nusinersen (Spinraza®) or onasemnogene abeparvovec (Zolgensma®), actively treated with risdiplam (Evrysdi®), and switching from Spinraza or Zolgensma to 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.
Conditions Studied
Study Locations (1)
Tennessee
- St. Jude Children's Research Hospital - Memphis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2025-10-29 |
| Est. Completion | 2027-03 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06532474
The ClinicalTrials.gov registry entry for NCT06532474 describes a study currently listed as recruiting, categorized as an unspecified phase. The registered enrollment target is 24 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 108-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (78% lower). The listed sponsor is St. Jude Children's Research Hospital, which has 410 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 0 interventions.
NCT06532474 reports 1 study location spanning 1 distinct geographic area - top geographies include Tennessee.
Frequently Asked Questions
What is clinical trial NCT06532474 about?
NCT06532474 is a clinical study titled "Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies". 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...
What is the current status of trial NCT06532474?
This trial is currently recruiting. The enrollment target is 24 participants. The study started on 2025-10-29. Estimated completion is 2027-03.
What conditions does trial NCT06532474 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy.
Who is sponsoring clinical trial NCT06532474?
This trial is sponsored by St. Jude Children's Research Hospital, which has 410 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06532474 being conducted?
This trial has 1 study location across Tennessee. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Read our methodology - how this data is sourced, computed, and verified.
Related
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