A Pilot Study of Biomarkers for Spinal Muscular Atrophy

Status: Completed
Location: See all (18) locations...
Study Type: Observational
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Maximum Age: 12
Healthy Volunteers: Accepts Healthy Volunteers
View:

• Age 2 to 12 years, inclusive

• In good health (other than SMA) in the judgement of the clinical investigator ar the time of assessment

Locations
United States
Alabama
University of Alabama at Birmingham
Birmingham
California
Stanford University
Stanford
Colorado
The Children's Hospital
Aurora
Iowa
University of Iowa
Iowa City
Massachusetts
Children's Hospital Boston
Boston
Maryland
Johns Hopkins Hospital
Baltimore
Michigan
Children's Hospital of Michigan, Detroit
Detroit
Minnesota
Mayo Clinic Rochester
Rochester
Missouri
Washington University Medical School
St. Louis
New York
Columbia University SMA Clinical Research Center
New York
Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati
The Ohio State University
Columbus
Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia
Texas
Children's Medical Center - Dallas
Dallas
Utah
University of Utah
Salt Lake City
Wisconsin
University of Wisconsin Hospital and Clinics
Madison
Other Locations
Canada
Children's Hospital - London Health Sciences Center
London
The Hospital for Sick Children
Toronto
Time Frame
Start Date: October 2008
Completion Date: March 2009
Participants
Target number of participants: 130
Treatments
SMA cohort
Subjects between the ages of 2-12 years diagnosed with SMA Type I, II, or III.
Control cohort
Healthy children between the ages of 2-12 years. These children may be either genetically-related siblings of SMA children (genetically confirmed non-carriers of SMA),or unrelated children.
Sponsors
Collaborators: The Spinal Muscular Atrophy Foundation
Leads: HealthCore-NERI

This content was sourced from clinicaltrials.gov

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