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Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy

NCT05518773 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 34

Last updated 2025-12-03

No results posted yet for this study

Summary

This study will focus on the pathophysiological underpinnings of reduced exercise capacity and fatigue in ambulatory patients with spinal muscular atrophy (SMA). There has been laboratory evidence to suggest that the molecular mechanisms underlying mitochondrial biogenesis may be vulnerable to survival motor neuron (SMN) protein deficiency. This is an observational, single visit study including 34 ambulatory SMA patients treated with SMN repletion therapies (risdiplam or nusinersen) for at least 6 months at enrollment.

Conditions

Interventions

OTHER

Observational

Observational

Sponsors & Collaborators

Principal Investigators

  • Jacqueline Montes, PT, EdD · Columbia University

Eligibility

Min Age
8 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-12-15
Primary Completion
2025-11-14
Completion
2025-11-14

Countries

  • United States

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05518773 on ClinicalTrials.gov