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Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development

NCT01736553 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 53

Last updated 2018-05-04

Study results available
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Summary

Spinal muscular atrophy (SMA) is the leading genetic cause of death of infants. Strong preclinical evidence suggests that effective therapy must be delivered as early as possible to prevent progression of the disease. The primary study objective will be to identify prognostic and surrogate biomarkers of disease progression that will facilitate the execution of therapeutic SMA clinical trials in infants.

Conditions

  • Spinal Muscular Atrophy (SMA)

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • Cure SMA

    collaborator OTHER

  • Massachusetts General Hospital

    collaborator OTHER

  • University of Iowa

    collaborator OTHER

  • Ohio State University

    lead OTHER

Principal Investigators

  • Stephen J Kolb, MD PhD · Ohio State University

Eligibility

Max Age
6 Months
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2012-12-31
Primary Completion
2015-09-30
Completion
2015-09-30

Countries

  • United States

Study Locations

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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 NCT01736553 on ClinicalTrials.gov