NCT07883655 Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMA
| NCT ID | NCT07883655 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Istituto Giannina Gaslini |
| Condition | Spinal Muscular Atrophy (SMA) |
| Study Type | OBSERVATIONAL |
| Enrollment | 200 participants |
| Start Date | 2025-02-05 |
| Primary Completion | 2026-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 200 participants in total. It began in 2025-02-05 with a primary completion date of 2026-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: * 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. Exclusion Criteria: * Presence of other neuromuscular or neurological diseases that could influence the clinical phenotype and biomarker profile. * Participation in other clinical trials involving investigational medicinal products for SMA within the 6 months prior to enrolment. * Presence of medical or psychological conditions that could compromise patient compliance or study safety. * Refusal to participate in the study or to provide informed consent.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07883655 clinical trial?
This trial is open to participants of all sexes, studying Spinal Muscular Atrophy (SMA). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07883655 currently recruiting?
Yes, NCT07883655 is actively recruiting participants. Contact the research team at claudiobruno@gaslini.org for enrollment information.
Where is the NCT07883655 trial being conducted?
This trial is being conducted at Genova, Italy, Milan, Italy, Naples, Italy.
Who is sponsoring the NCT07883655 clinical trial?
NCT07883655 is sponsored by Istituto Giannina Gaslini. The trial plans to enroll 200 participants.