A Pilot Study of Biomarkers for Spinal Muscular Atrophy
Public ClinicalTrials.gov record NCT00756821. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT00756821
- Recruitment status
- Completed
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 130 participants
Conditions and interventions
Conditions
Interventions
Not listed
Eligibility (public fields only)
- Age range
- 2 Years to 12 Years
- Sex
- All
- Healthy volunteers
- Accepts healthy volunteers
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 2008 (Type not available)
- Primary completion
- Mar 2009 (Actual)
- Completion
- Mar 2009 (Actual)
- Last update posted
- Oct 24, 2012
2008 – 2009
United States locations
- U.S. sites
- 16
- U.S. states
- 15
- U.S. cities
- 16
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | — |
| Stanford University | Stanford | California | 94305 | — |
| The Children's Hospital | Aurora | Colorado | 80045 | — |
| University of Iowa | Iowa City | Iowa | 52242 | — |
| Johns Hopkins Hospital | Baltimore | Maryland | 21287 | — |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | — |
| Children's Hospital of Michigan, Detroit | Detroit | Michigan | 48201 | — |
| Mayo Clinic Rochester | Rochester | Minnesota | 55905 | — |
| Washington University Medical School | St Louis | Missouri | 63110 | — |
| Columbia University SMA Clinical Research Center | New York | New York | 10032 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| The Ohio State University | Columbus | Ohio | 43210 | — |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Children's Medical Center - Dallas | Dallas | Texas | 75207 | — |
| University of Utah | Salt Lake City | Utah | 84132 | — |
| University of Wisconsin Hospital and Clinics | Madison | Wisconsin | 53792 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 2 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00756821, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 24, 2012 · Synced Oct 23, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT00756821 live on ClinicalTrials.gov.