Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
A Pilot Study of Biomarkers for Spinal Muscular Atrophy
NCT00756821 · View on ClinicalTrials.gov ↗
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 Studied
Study Locations (18)
Ohio
- Cincinnati Children's Hospital Medical Center — Cincinnati
- The Ohio State University — Columbus
Ontario
- Children's Hospital - London Health Sciences Center — London
- The Hospital for Sick Children — Toronto
Alabama
- University of Alabama at Birmingham — Birmingham
California
- Stanford University — Stanford
Colorado
- The Children's Hospital — Aurora
Iowa
- University of Iowa — Iowa City
Maryland
- Johns Hopkins Hospital — Baltimore
Massachusetts
- Children's Hospital Boston — Boston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 130 participants |
| Start Date | 2008-10 |
| Est. Completion | 2009-03 |
Interested in This Trial?
Always speak with your doctor before enrolling in a clinical trial.
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00756821
The ClinicalTrials.gov registry entry for NCT00756821 describes a study currently listed as completed. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 130 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Carelon Research, which has 10 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT00756821 reports 18 study locations spanning 16 distinct geographic areas — top geographies include Ohio, Ontario, Alabama. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT00756821 about?
NCT00756821 is a clinical study titled "A Pilot Study of Biomarkers for Spinal Muscular Atrophy". 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, bo...
What is the current status of trial NCT00756821?
This trial is currently completed. The enrollment target is 130 participants. The study started on 2008-10. Estimated completion is 2009-03.
What conditions does trial NCT00756821 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT00756821?
This trial is sponsored by Carelon Research, which has 10 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT00756821 being conducted?
This trial has 18 study locations across Alabama, California, Colorado, Iowa, Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How Clinical Trials Work
Understand phases 1-4, trial design, randomization, and the informed consent process.
Patient Rights in Clinical Trials
Your rights as a participant: consent, withdrawal, privacy, and who to contact.
Finding the Right Clinical Trial
A practical guide to searching trials, understanding eligibility, and evaluating options.
All Guides
Browse our complete library of clinical trial educational resources.
Read our methodology — how this data is sourced, computed, and verified.