NCT05768048Recruiting
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
SMA Natural History Study Evaluating Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
Source: ClinicalTrials.gov · Last updated 14 Mar 2023 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 500 (estimated)
- First submitted
- 2 Mar 2023
- Start date
- 28 Nov 2022
- Primary completion date
- 27 Nov 2027
- Lead sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT05768048
What you commit to
- Study type: Observational
- Planned participants: 500
- From 28 Nov 2022 to the primary completion date, 27 Nov 2027
- 5 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.
Other
- unable to proviude consent
Who the study is looking for
- all patients with mutationsin the SMAN1 gene
Sex: All · Does not accept healthy volunteers
Sites (5)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Istituto Gaslini Coordinator of this site? Confirm its status | Genova | Italy | Recruiting |
| Nemo Sud Coordinator of this site? Confirm its status | Messina | Italy | Recruiting |
| Centro Clinico Nemo Coordinator of this site? Confirm its status | Milan | Italy | Recruiting |
| Policlinico Gemelli Coordinator of this site? Confirm its status | Rome | Italy | Recruiting |
| Ospedale Bambino gesu Coordinator of this site? Confirm its status | Rome | Italy | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.