Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments

NCT05768048 · tracked via the Priya Life Science Italy tracker

Sponsor

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Phase

Observational

Started

2022-11-28

Last updated

2023-03-14

Condition(s) studied

Spinal Muscular Atrophy

Study 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

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

Inclusion Criteria: all patients with mutationsin the SMAN1 gene Exclusion Criteria: unable to proviude consent

Primary outcome measure(s)

motor function using the HFMSE (min score 0, max 74 indicating best performance) — 5 years
motor scale

Trial sites (5)

Facility	City	Region	Status
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

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Other trials for the same condition

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab Active, not recruiting

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy Recruiting

A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) Active, not recruiting

A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05768048 on ClinicalTrials.gov ↗ ← All trials in Italy