NCT07883655Recruiting
Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMA
Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMA - Approcci Avanzati Multi-omici e Sistemici Per Identificare Nuovi Biomarcatori Per la SMA
Source: ClinicalTrials.gov · Last updated 20 Oct 2026
Brief summary (registry, in English)
Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder caused by mutations in the SMN1 gene, leading to motor neuron degeneration and progressive muscle atrophy. Despite recent advances in therapeutic options, the clinical management of SMA remains challenging due to the heterogeneity of disease progression and variability in treatment response. This study aims to identify and validate novel biomarkers to improve the diagnosis, prognosis, and therapeutic monitoring of patients with SMA through a multi-omics and translational research approach.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 200 (estimated)
- First submitted
- 9 Oct 2026
- Start date
- 5 Feb 2025
- Primary completion date
- Dec 2026
- Lead sponsor
- Istituto Giannina Gaslini
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT07883655
What you commit to
- Study type: Observational
- Planned participants: 200
- From 5 Feb 2025 to the primary completion date, Dec 2026
- 3 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Participation in other clinical trials involving investigational medicinal products for SMA within the 6 months prior to enrolment.
Other illness
- Presence of other neuromuscular or neurological diseases that could influence the clinical phenotype and biomarker profile.
- Presence of medical or psychological conditions that could compromise patient compliance or study safety.
Other
- Refusal to participate in the study or to provide informed consent.
Who the study is looking for
- Genetically confirmed diagnosis of SMA, with homozygous deletion or mutation of the SMN1 gene, for symptomatic patients.
- Diagnosis of SMA through newborn screening, with genetic confirmation, for newborns.
- Ability of parents/legal guardians to provide informed consent for minor patients.
- Willingness to undergo the study procedures, including blood sampling and collection of biological specimens.
Sex: All · Accepts healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| IRCCS Istituto Giannina Gaslini Coordinator of this site? Confirm its status | Genova | Italy | Recruiting |
| Fondazione Ca' Granda Ospedale Maggiore Policlinico Coordinator of this site? Confirm its status | Milan | Italy | Recruiting |
| Istituto di Bioscienze e Biorisorse IBBR Coordinator of this site? Confirm its status | Naples | Italy | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.