Prospective Evaluation of Infants With Spinal Muscular Atrophy:

Status unconfirmed

Conditions studied: Spinal Muscular Atrophy

In brief

SPOT SMA is a prospective NIH-supported clinical study targeting pre-symptomatic or recently diagnosed infants and children with Spinal Muscular Atrophy (SMA) types 1, 2, or 3 and their healthy control siblings less than 36 months of age at the time of study enrollment. The main objective of the study is to prospectively collect longitudinal clinical outcomes and provide counseling and education to parents of newly diagnosed children. The study will assess the impact of current standard of care management paradigms and interventions on health outcomes in newly diagnosed SMA infants and children with type 1, 2 or 3 and age appropriate controls. There is no investigational drug and no specific intervention in this study. Rather, the investigators will document outcomes related to current therapies provided to participating subjects, and will educate participants about possible clinical trial opportunities.

Key facts

Study ID

NCT02831296

Run by

Massachusetts General Hospital

People needed

1000

Starts

2016-02-01

Last updated by the study team

2020-09-02

Who can join

Age: any. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• For affected subjects: genetic diagnosis of SMA
• For unaffected family members: parent or sibling of any age (without genetic diagnosis of SMA) of affected subject enrolled in study

You may not qualify if…

• None

Where it is running

• Massachusetts General Hospital — Boston, Massachusetts, United States (enrolling)

Full record on ClinicalTrials.gov

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