NCT05768048 Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
| NCT ID | NCT05768048 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Fondazione Policlinico Universitario Agostino Gemelli IRCCS |
| Condition | Spinal Muscular Atrophy |
| Study Type | OBSERVATIONAL |
| Enrollment | 500 participants |
| Start Date | 2022-11-28 |
| Primary Completion | 2027-11-27 |
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 500 participants in total. It began in 2022-11-28 with a primary completion date of 2027-11-27.
⚠ 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
This is an observational multicenter retrospective and prospective study on natural history of SMA also considering the 'new natural history' secondary to the availability of commercially available therapies. All the patients enrolled to date in the Italian registry, if not part of clinical trials, will be included in the present study.
Eligibility Criteria
Inclusion Criteria: all patients with mutationsin the SMAN1 gene Exclusion Criteria: unable to proviude consent
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05768048 clinical trial?
This trial is open to participants of all sexes, studying Spinal Muscular Atrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05768048 currently recruiting?
Yes, NCT05768048 is actively recruiting participants. Contact the research team at eugeniomaria.mercuri@policlinicogemelli.it for enrollment information.
Where is the NCT05768048 trial being conducted?
This trial is being conducted at Genova, Italy, Messina, Italy, Milan, Italy, Rome, Italy and 1 additional location.
Who is sponsoring the NCT05768048 clinical trial?
NCT05768048 is sponsored by Fondazione Policlinico Universitario Agostino Gemelli IRCCS. The trial plans to enroll 500 participants.