Machine-learning algorithm to predict treatment response in Spinal Muscular Atrophy
MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy
This study is testing a new computer program that uses patient information to see if it can predict how well different treatments will work for people with Spinal Muscular Atrophy.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 247 (estimated) |
| Sex | All |
| Sponsor | Fondazione Policlinico Universitario Agostino Gemelli IRCCS Academic / other |
| Locations | 1 site (Roma) |
| Trial ID | NCT05769465 on ClinicalTrials.gov |
What this trial studies
This project aims to collect clinical data and patient-reported outcomes from individuals with Spinal Muscular Atrophy (SMA) who are receiving approved therapies. By identifying novel biomarkers and RNA molecular signatures, the study will develop a machine-learning algorithm to predict therapeutic responses based on individual patient characteristics and clinical outcomes. The goal is to enhance patient stratification and guide personalized treatment approaches for SMA. This observational study will leverage artificial intelligence methodologies to improve understanding of treatment variability among patients.
Who should consider this trial
Good fit: Ideal candidates include individuals with a confirmed genetic diagnosis of SMA and clinical phenotypes of type I, II, or III.
Not a fit: Patients without a confirmed genetic diagnosis of SMA or those with other types of SMA may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to more personalized and effective treatment strategies for patients with Spinal Muscular Atrophy.
How similar studies have performed: While there have been studies exploring biomarkers in SMA, the specific use of machine learning for predicting treatment response is a novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * confirmed genetic diagnosis of SMA (5q) * clinical phenotype of type I or II or III; * able to provide (patient/caregiver) written informed consent Exclusion Criteria: * None
Where this trial is running
Roma
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Roma, Italy (Recruiting)
Study contacts
- Principal investigator: Giorgia Coratti, PhD — Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Study coordinator: Comitato Etico
- Email: comitato.etico@policlinicogemelli.it
- Phone: 0630156124
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.