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Related Experiment Video

Updated: Feb 9, 2026

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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Evaluator Training and Reliability for SMA Global Nusinersen Trials1.

Allan M Glanzman1, Elena S Mazzone2, Sally Dunaway Young3,4

  • 1Department of Physical Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA.

Journal of Neuromuscular Diseases
|June 6, 2018
PubMed
Summary

Rigorous training for evaluators in nusinersen clinical trials ensured reliable outcome measures. This methodology achieved excellent reliability for key assessments in spinal muscular atrophy (SMA) studies.

Keywords:
SMAmulticenternusinersenoutcome assessmentoutcome measuresreliability of resultsspinal muscular atrophystudies

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Area of Science:

  • Neurology
  • Clinical Trials Methodology
  • Biostatistics

Background:

  • Nusinersen clinical trials require reliable outcome measures for assessing spinal muscular atrophy (SMA).
  • Key outcome measures included the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), Hammersmith Functional Motor Scale Expanded (HFMSE), and Revised Upper Limb (RULM).

Purpose of the Study:

  • To establish and validate a training methodology for optimizing the reliability of outcome measures in nusinersen clinical trials.
  • To ensure consistent and accurate assessments across multiple sites and evaluators for pediatric neuromuscular disorders.

Main Methods:

  • Implemented a comprehensive training program involving video review, quarterly conference calls, and item scoring checks to ensure evaluator competence.
  • Established intra- and inter-rater reliability through baseline assessments, screening, and ongoing video review processes.
  • Utilized Intraclass Correlation Coefficients (ICC) to quantify reliability across initial training and annual retraining sessions.

Main Results:

  • Achieved excellent inter- and intra-rater reliability, with ICCs ranging from 0.906-0.994 for initial training and 0.824-0.996 for retraining.
  • Demonstrated high reliability for specific scales: CHOP INTEND (ICC = 0.824-0.951), HFMSE (ICC = 0.981-0.996), and RULM (ICC = 0.966-0.990).
  • Specific intra-rater reliability values were reported for CHOP INTEND (ICC = 0.895), HFMSE (ICC = 0.959), and RULM (ICC = 0.948).

Conclusions:

  • A rigorous evaluator training methodology is crucial for ensuring the reliability of assessments in multicenter international clinical trials for spinal muscular atrophy (SMA).
  • The established training protocols effectively support reliable data collection for critical outcome measures in SMA research.