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Prospective Evaluation of Infants With Spinal Muscular Atrophy:

NCT02831296 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 1000

Last updated 2020-09-02

No results posted yet for this study

Summary

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.

Conditions

Sponsors & Collaborators

  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

    collaborator NIH

  • University of Utah

    collaborator OTHER

  • University of Massachusetts, Worcester

    collaborator OTHER

  • ARUP Laboratories

    collaborator OTHER

  • Newborn Screening Translational Research Network

    collaborator UNKNOWN

  • American College of Medical Genetics and Genomics

    collaborator UNKNOWN

  • Children's Hospital Medical Center, Cincinnati

    collaborator OTHER

  • Massachusetts General Hospital

    lead OTHER

Principal Investigators

  • Kathryn J Swoboda, MD · Massachusetts General Hospital

Eligibility

Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2016-02-29
Primary Completion
2022-03-31

Countries

  • United States

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02831296 on ClinicalTrials.gov