Status:
UNKNOWN
Natural History of Types 2 and 3 SMA in Taiwan
Lead Sponsor:
Kaohsiung Medical University Chung-Ho Memorial Hospital
Collaborating Sponsors:
Biogen
National Taiwan University Hospital
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
6-70 years
Brief Summary
The primary objective of this study is to investigate the natural history of spinal muscular atrophy (SMA) types 2 and 3 patients in Taiwan. This study will provide further insights into the clinical ...
Detailed Description
As with other rare diseases, individual groups of SMA have therefore opted to share patient information in the form of clinical sites to increase the overall patient cohorts on which clinical outcomes...
Eligibility Criteria
Inclusion
- Patients are diagnosed with SMA types 2 or 3
- Generalized hypotonia and muscle weakness, weakness of the legs is greater than the arms, and the proximal part is weaker than distal part of extremities.
- SMN1 gene deletion or mutation and/or neurogenic changes in electromyogram and/or muscle pathology.
Exclusion
- Non-5q SMA (no deletion or mutation of SMN1 gene) patients.
- Type 1 SMA patients.
Key Trial Info
Start Date :
September 1 2017
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 1 2019
Estimated Enrollment :
300 Patients enrolled
Trial Details
Trial ID
NCT03300869
Start Date
September 1 2017
End Date
December 1 2019
Last Update
July 26 2019
Active Locations (1)
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1
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, Taiwan, 807