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A Pilot Study of Biomarkers for Spinal Muscular Atrophy

NCT00756821 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 130

Last updated 2012-10-24

No results posted yet for this study

Summary

The goal of this pilot study is to identify a marker or panel of markers in the blood or urine from a wide range of Spinal Muscular Atrophy (SMA) patients that segregates with measures of clinical severity. From this identification of candidate biomarkers, it is hoped that further investigations, both longitudinal natural history and clinical efficacy studies, will verify a biomarker with the sensitivity and specificity that will allow its eventual use as a validated pharmacodynamic marker or surrogate endpoint. In addition, this effort may elucidate biological pathways that may be potential therapeutic targets.

Conditions

Sponsors & Collaborators

  • The Spinal Muscular Atrophy Foundation

    collaborator OTHER

  • Carelon Research

    lead OTHER

Principal Investigators

  • Richard Finkel, MD · Children's Hospital of Philadelphia

  • Thomas Crawford, MD · Johns Hopkins University

  • Petra Kaufmann, MD · Columbia University

Eligibility

Min Age
2 Years
Max Age
12 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2008-10-31
Primary Completion
2009-03-31
Completion
2009-03-31

Countries

  • United States
  • Canada

Study Locations

More Related Trials

Entities

Diseases

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 NCT00756821 on ClinicalTrials.gov