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NCT00756821 · ClinicalTrials.gov registry record

A Pilot Study of Biomarkers for Spinal Muscular Atrophy

A clinical trial of Spinal Muscular Atrophy, sponsored by Carelon Research.

Completed
Registry status
130
Enrollment target
18
Study locations

NCT00756821: Completed study of Spinal Muscular Atrophy, sponsored by Carelon Research.

NCT00756821 is a study of Spinal Muscular Atrophy that has completed, run by Carelon Research. The registered enrollment target is 130 participants, above the 103-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (26% higher). The trial reports 18 study locations across 16 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT00756821, a study of Spinal Muscular Atrophy, has completed, sponsored by Carelon Research.

COMPLETED
Registry status
130 participants
Enrollment target
18
Study locations

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

FieldValue
Enrollment Target 130 participants
Start Date 2008-10
Est. Completion 2009-03
Carelon Research

50 total trials

What the finished NCT00756821 record still lists

NCT00756821 is an observational study that tracks outcomes without assigning an intervention. The registered 130 participants enrollment target is mid-sized for trials with a published cap, above the 103-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (26% higher).

The record links to 1 condition, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 0 interventions.

NCT00756821 lists 18 locations in 16 states (Ohio, Ontario, Alabama).

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.

Who is sponsoring clinical trial NCT00756821?

This trial is sponsored by Carelon Research, which has 50 total clinical trials registered on ClinicalTrials.gov.

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.

Similar trials for Spinal Muscular Atrophy

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT00756821's enrollment target sits among peer trials

130 8th of 22 higher than 15 of 22 other Spinal Muscular Atrophy trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Spinal Muscular Atrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT00756821, the US trial registry maintained by the National Library of Medicine. NCT00756821 (mid enrollment · multi site footprint · completed) retrieved and formatted by PlainTrial, see methodology.