A Pilot Study of Biomarkers for Spinal Muscular Atrophy

Completed

Conditions studied: Spinal Muscular Atrophy

In brief

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.

Key facts

Study ID

NCT00756821

Run by

Carelon Research

People needed

130

Starts

2008-10-01

Expected to finish

2009-03-01

Last updated by the study team

2012-10-24

Who can join

Age: 2 and older, up to 12. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• Age 2 to 12 years, inclusive
• In good health (other than SMA) in the judgement of the clinical investigator ar the time of assessment

You may not qualify if…

• Systemic or specific-organ illness
• Any known genetic condition other than SMA requiring pharmaceutical treatment
• Use of any putative SMN-enhancing medications or treatments in the past 14 days prior to enrollment
• Use of carnitine, creatine, oral albuterol or riluzole for 14 days prior to enrollment
• Use of any oral prescription medications for 14 days prior to enrollment (exceptions: anti-reflux medications, constipation or stoll softening medications, stool bulking agents, and inhaled bronchodilator medications)
• Any illness requiring treatment of antibiotics or anti-inflammatory medication within the past 14 days
• Any rash requiring treatment within the past 7 days
• Any severe asthma attack requiring treatment with oral or parenteral steroids within the past 7 days
• Any fever over 100 degrees Fahrenheit or 38 degree Celsius within the past 7 days
• Any immunization within the past 7 days
• Any injury sustained that resulted in a bone fracture or needed stitches within the past 7 days
• Any surgery within the past 7 days
• Any receipt of anesthesia within the past 7 days
• Any Emergency Room visit or hospitalization within the past 7 days
• Any stomach illness with vomiting within the past 7 days
• Any migraine headache within the past 7 days
• Participation in a clinical trial (except observational studies) within the past 7 days

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• Stanford University — Stanford, California, United States
• The Children's Hospital — Aurora, Colorado, United States
• University of Iowa — Iowa City, Iowa, United States
• Johns Hopkins Hospital — Baltimore, Maryland, United States
• Children's Hospital Boston — Boston, Massachusetts, United States
• Children's Hospital of Michigan, Detroit — Detroit, Michigan, United States
• Mayo Clinic Rochester — Rochester, Minnesota, United States
• Washington University Medical School — St Louis, Missouri, United States
• Columbia University SMA Clinical Research Center — New York, New York, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• The Ohio State University — Columbus, Ohio, United States
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children's Medical Center - Dallas — Dallas, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• University of Wisconsin Hospital and Clinics — Madison, Wisconsin, United States
• Children's Hospital - London Health Sciences Center — London, Ontario, Canada
• The Hospital for Sick Children — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.