Status:
COMPLETED
Two Different Treatment Modalities in Patients With Spinal Muscular Atrophy
Lead Sponsor:
Istanbul Medipol University Hospital
Collaborating Sponsors:
The Scientific and Technological Research Council of Turkey
Conditions:
Muscular Atrophy, Spinal
Eligibility:
All Genders
5-18 years
Phase:
NA
Brief Summary
Spinal Muscular Atrophy (SMA) is a severe neuromuscular disorder characterized by the degeneration of alpha motor neurons in the spinal cord, resulting in progressive muscle atrophy and weakness, part...
Detailed Description
Spinal Muscular Atrophy (SMA) is a severe neuromuscular disease characterized by degenerating alpha motor neurons in the spinal cord, resulting in progressive and predominantly proximal and axial musc...
Eligibility Criteria
Inclusion
- Those with genetic documentation of 5q spinal muscular atrophy (SMA) homozygous gene deletion, mutation, or compound heterozygote and who have been clinically diagnosed with SMA,
- Those between the ages of 5-18,
- Maximum inspiratory capacity is less than 60 cmH2O,
- Children who can sit unsupported for at least 5 seconds and who have no or 3 weeks of acute reversible events affecting the upper respiratory tract.
Exclusion
- Having had upper extremity and spine surgery,
- Having other orthopedic and neurological problems,
- Having cognitive impairments that may prevent understanding simple verbal commands,
- Having visual or auditory disabilities,
- Premature birth
Key Trial Info
Start Date :
February 21 2024
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 29 2024
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT06178653
Start Date
February 21 2024
End Date
December 29 2024
Last Update
May 2 2025
Active Locations (1)
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1
Medipol Mega University Hospital
Istanbul, Bağcılar, Turkey (Türkiye), 34214