MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy
NCT ID: NCT05769465
Last Updated: 2023-09-13
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
247 participants
OBSERVATIONAL
2023-04-01
2026-04-01
Brief Summary
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The aim of this project is:
* collect clinical data and patient-reported outcome measures (PROM) from patients treated with nusinersen, risdiplam, onasemnogene abeparvovec,
* identify novel biomarkers and RNA molecular signature profiling,
* develop a predictive algorithm using artificial intelligence (AI) methodologies based on machine learning (ML), able to integrate clinical outcomes, patients' characteristics, and specific biomarkers.
This effort will help to better stratify the SMA patients and to predict their therapeutic outcome, thus to address patients towards personalized therapies.
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Detailed Description
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Conditions
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Study Design
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COHORT
OTHER
Study Groups
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Patients treated with nusinersen
disease modifying treatments
Patients will be enrolled if exposed to nusinersen, risdiplam, onasemnogene abeparvovec
Patients treated with risdiplam
disease modifying treatments
Patients will be enrolled if exposed to nusinersen, risdiplam, onasemnogene abeparvovec
Patients treated with onasemnogene abeparvovec
disease modifying treatments
Patients will be enrolled if exposed to nusinersen, risdiplam, onasemnogene abeparvovec
Patients naive from disease modifying treatments
No interventions assigned to this group
Interventions
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disease modifying treatments
Patients will be enrolled if exposed to nusinersen, risdiplam, onasemnogene abeparvovec
Eligibility Criteria
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Inclusion Criteria
* clinical phenotype of type I or II or III;
* able to provide (patient/caregiver) written informed consent
Exclusion Criteria
ALL
No
Sponsors
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
OTHER
Responsible Party
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Principal Investigators
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Giorgia Coratti, PhD
Role: PRINCIPAL_INVESTIGATOR
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Locations
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, , Italy
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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GR-2021-12374579
Identifier Type: OTHER_GRANT
Identifier Source: secondary_id
5488
Identifier Type: -
Identifier Source: org_study_id
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