Observing the natural history of spinal muscular atrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study aims to collect and analyze data on the natural history of spinal muscular atrophy (SMA) in a large cohort of patients across five academic centers in Italy. By combining retrospective and prospective data, the study seeks to identify factors influencing disease progression and describe patterns of functional measures, adverse events, and levels of care in both treated and untreated patients. The registry will also document epidemiological data and provide insights into supportive care and hospitalization experiences of SMA patients. Trained clinical evaluators and data managers will ensure the reliability of the data collected.

Why it matters

Who can join: the full rules

Inclusion Criteria:

all patients

Exclusion Criteria:

if enrolled in clinical trials

Where it's running

• Istituto Gaslini
  Genova, Italy Recruiting
• Nemo Sud
  Messina, Italy Recruiting
• Centro Clinico nemo
  Milan, Italy Recruiting
• Ospedale Bambino gesu
  Rome, Italy Recruiting
• Policlinico gemelli
  Rome, Italy Recruiting

Study contacts

• Principal investigator: Eugenio Mercuri, MD — F Policlinico Gemelli IRCCS
• Study coordinator: Eugenio Mercuri, MD
• Email: eugeniomaria.mercuri@policlinicogemelli.it
• Phone: 06 3015

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Salanersen after early gene therapy in babies with SMA

  This study tests whether adding salanersen (BIIB115) after early gene therapy with Onasemnogene abeparvovec helps babies with SMA.

  Recruiting Phase 3 Biogen · Flower Mound, Texas · NCT07444450

• Salanersen given before symptoms to help movement in babies with genetic SMA

  This study will try salanersen in newborns with genetically confirmed SMA who have 2 or 3 copies of SMN2 to see if starting treatment before symptoms prevents or lessens movement problems.

  Recruiting at 14 sites Phase 3 Biogen · Chicago, Illinois and 13 other locations · NCT07221669

• Understanding how nusinersen is processed in the body using a new delivery system for spinal muscular atrophy

  This study is testing a new way to deliver the medication nusinersen for spinal muscular atrophy to see how it works in the body compared to the usual method.

  Recruiting at 19 sites Phase 1 Biogen · Phoenix, Arizona and 20 other locations · NCT06555419

• Evaluating pregnancy outcomes in women with spinal muscular atrophy exposed to Spinraza

  This study is trying to see how pregnancy goes for women with spinal muscular atrophy who have used Spinraza, compared to women without the condition who haven't used the drug.

  Recruiting at 13 sites Biogen · Palo Alto, California and 13 other locations · NCT05789758

• Pregnancy-time treatment of fetal SMA: a registry of mother-child outcomes

  This registry collects information on pregnancies where a fetus diagnosed with spinal muscular atrophy was treated with a disease‑modifying therapy in the womb to see how the pregnancy and baby fare after birth.

  Recruiting St. Jude Children's Research Hospital · Memphis, Tennessee · NCT07805278

• Intrathecal Zolgensma in spinal muscular atrophy patients in Japan

  This study tests the safety and real‑world effectiveness of intrathecal onasemnogene abeparvovec in SMA patients in Japan.

  Recruiting at 4 sites Novartis · Toyoake, Aichi-ken and 3 other locations · NCT07767890

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.