Development of a Multidisciplinary Network for Clinical and Laboratory Research for SMA
1 other identifier
observational
400
0 countries
N/A
Brief Summary
The goal of this observational study is to to establish profiles of clinical progression in patients affected by the different types of SMA (type I, II and III) treated with the currently approved drugs using a structured battery of clinical tests. Another goal of the study is to assess the progression of the disease in patients identified through neonatal screening.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jun 2023
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 18, 2023
CompletedFirst Posted
Study publicly available on registry
May 19, 2023
CompletedStudy Start
First participant enrolled
June 30, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
June 30, 2025
CompletedMay 19, 2023
April 1, 2023
1 year
April 18, 2023
May 10, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
Establish profiles of clinical progression in patients affected by the different types of SMA (type I, II and III) treated with the currently approved drugs using a structured battery of clinical tests.
2-year
Assess the progression of the disease in patients identified through neonatal screening
2-year
Identification of molecular biomarkers that associate with disease course and response to therapies
2-year
Eligibility Criteria
Patients with spinal muscular atrophy regardless of age, type of SMA or treatment
You may qualify if:
- All SMA patients
You may not qualify if:
- Inability to understand or to provide informed consent.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 18, 2023
First Posted
May 19, 2023
Study Start
June 30, 2023
Primary Completion
June 30, 2024
Study Completion
June 30, 2025
Last Updated
May 19, 2023
Record last verified: 2023-04