Robust Markers and Sample Sizes for Multicenter Trials of Huntington Disease

The identification of sensitive biomarkers is essential to validate therapeutics for Huntington disease (HD). We directly compare structural imaging markers across the largest collective imaging HD dataset to identify a set of imaging markers robust to multicenter variation and to derive upper estimates on sample sizes for clinical trials in HD.

• Publication year

  2020

• Venue

  Annals of Neurology

• Publication date

  2020-02-27

• Fields of study

  Medicine

• Identifiers
  DOI 10.1002/ana.25709PMID 32105364PMCID 7187160
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

• Huntington’s Disease Clinical Trials Corner: August 2018

  2018cited by this paper
• Deep gray matter volume loss drives disability worsening in multiple sclerosis

  2018cited by this paper
• An image‐based model of brain volume biomarker changes in Huntington's disease

  2018cited by this paper
• Reduced amygdala volumes are related to motor and cognitive signs in Huntington's disease: The IMAGE-HD study

  2018cited by this paper
• Neurofilament light protein in blood as a potential biomarker of neurodegeneration in Huntington's disease: a retrospective cohort analysis

  2017cited by this paper
• Motor, cognitive, and functional declines contribute to a single progressive factor in early HD

  2017cited by this paper
• Imaging plus X: multimodal models of neurodegenerative disease

  2017cited by this paper
• Deep grey matter volume loss drives disability worsening in multiple sclerosis

  2017cited by this paper
• The role of the human globus pallidus in Huntington's disease

  2016cited by this paper
• Symbol Digit Modalities Test

  2016cited by this paper
• MRI Segmentation of the Human Brain: Challenges, Methods, and Applications

  2015cited by this paper
• Short-interval observational data to inform clinical trial design in Huntington's disease

  2015cited by this paper
• Geodesic Information Flows: Spatially-Variant Graphs and Their Application to Segmentation and Fusion

  2015cited by this paper
• Clinical and Biomarker Changes in Premanifest Huntington Disease Show Trial Feasibility: A Decade of the PREDICT-HD Study

  2014cited by this paper
• Visual system integrity and cognition in early Huntington's disease

  2014cited by this paper
• Fitting Linear Mixed-Effects Models Using lme4

  2014cited by this paper
• R: A language and environment for statistical computing.

  2014cited by this paper
• Predictors of phenotypic progression and disease onset in premanifest and early-stage Huntington's disease in the TRACK-HD study: analysis of 36-month observational data.

  2013cited by this paper
• Multi-Modal Neuroimaging in Premanifest and Early Huntington’s Disease: 18 Month Longitudinal Data from the IMAGE-HD Study

  2013cited by this paper
• Functional and connectivity changes during working memory in Huntington's disease: 18 month longitudinal data from the IMAGE-HD study.

  2013cited by this paper
• Structural MRI in Huntington's disease and recommendations for its potential use in clinical trials.

  2013cited by this paper
• Potential endpoints for clinical trials in premanifest and early Huntington's disease in the TRACK-HD study: analysis of 24 month observational data.

  2012cited by this paper
• Basal ganglia atrophy in prodromal Huntington's disease is detectable over one year using automated segmentation

  2011cited by this paper
• Development of biomarkers for Huntington's disease.

  2011cited by this paper
• Striatal and white matter predictors of estimated diagnosis for Huntington disease.

  2010cited by this paper
• Early atrophy of pallidum and accumbens nucleus in Huntington’s disease

  2010cited by this paper
• Cerebral cortex structure in prodromal Huntington disease.

  2010cited by this paper
• Data Analysis Using Regression and Multilevel/Hierarchical Models

  2009cited by this paper
• Prefrontal cortex volume reduction on MRI in preclinical Huntington's disease relates to visuomotor performance and CAG number.

  2009cited by this paper
• Biological and clinical manifestations of Huntington's disease in the longitudinal TRACK-HD study: cross-sectional analysis of baseline data.

  2009cited by this paper
• Detection of Huntington’s disease decades before diagnosis: the Predict-HD study

  2007cited by this paper
• Distribution of grey matter atrophy in Huntington's disease patients: A combined ROI-based and voxel-based morphometric study

  2006cited by this paper
• Measurement error of two different techniques for brain atrophy assessment in multiple sclerosis

  2004cited by this paper
• A new model for prediction of the age of onset and penetrance for Huntington's disease based on CAG length

  2004cited by this paper
• Onset and rate of striatal atrophy in preclinical Huntington disease

  2004cited by this paper
• An introduction to power and sample size estimation

  2003cited by this paper
• The distribution of structural neuropathology in pre-clinical Huntington's disease.

  2002cited by this paper
• Statistical power and optimal design for multisite randomized trials.

  2000cited by this paper
• Frontal lobe volume in patients with Huntington's disease

  1998cited by this paper
• CAG repeat number governs the development rate of pathology in Huntington's disease

  1997cited by this paper
• Longitudinal change in basal ganglia volume in patients with Huntington's disease

  1997cited by this paper
• Unified Huntington's disease rating scale: Reliability and consistency

  1996cited by this paper
• Half a century of research on the Stroop effect: an integrative review.

  1991cited by this paper
• Statistical Power Analysis for the Behavioral Sciences

  1969cited by this paper

• Shape matters: Predicting Huntington's disease using progression modelling

  2026cites this paper
• MIMI-ONET: Multi-Modal image augmentation via Butterfly Optimized neural network for Huntington DiseaseDetection.

  2025cites this paper
• Brain Age as a New Measure of Disease Stratification in Huntington's Disease

  2025cites this paper
• Optimizing Screening for Intrastriatal Interventions in Huntington's Disease Using Predictive Models

  2024influential citation
• Prognostic enrichment for early-stage Huntington’s disease: An explainable machine learning approach for clinical trial

  2024cites this paper
• Predicting Huntington's disease state with ensemble learning & sMRI: more than just the striatum

  2023cites this paper
• The shape of things to come. Mapping spatiotemporal progression of striatal morphology in Huntington disease: The IMAGE-HD study.

  2023influential citation
• Longitudinal imaging highlights preferential basal ganglia circuit atrophy in Huntington’s disease

  2023cites this paper
• The temporal event-based model: Learning event timelines in progressive diseases

  2023cites this paper
• Progression of atypical parkinsonian syndromes: PROSPECT-M-UK study implications for clinical trials

  2023cites this paper
• Longitudinal mapping of cortical surface changes in Huntington’s Disease

  2022cites this paper
• Predictive Modeling of Huntington's Disease Unfolds Thalamic and Caudate Atrophy Dissociation

  2022cites this paper
• Considerations for clinical trial design and conduct in the evaluation of novel advanced therapeutics in neurodegenerative disease.

  2022cites this paper
• What is the Pathogenic CAG Expansion Length in Huntington’s Disease?

  2021cites this paper
• Multimodal Imaging and Visual Evoked Potentials Reveal Key Structural and Functional Features That Distinguish Symptomatic From Presymptomatic Huntington's Disease Brain

  2021cites this paper
• Imaging in Huntington’s

  2021cites this paper
• Revealing the Timeline of Structural MRI Changes in Premanifest to Manifest Huntington Disease

  2021cites this paper
• A Multi-Study Model-Based Evaluation of the Sequence of Imaging and Clinical Biomarker Changes in Huntington’s Disease

  2021cites this paper
• Tracking Huntingtonʼs Disease Progression Using Motor, Functional, Cognitive, and Imaging Markers

  2021cites this paper
• Neuroimaging, Urinary, and Plasma Biomarkers of Treatment Response in Huntington’s Disease: Preclinical Evidence with the p75NTR Ligand LM11A-31

  2021cites this paper
• hiPSCs for predictive modelling of neurodegenerative diseases: dreaming the possible

  2021cites this paper
• Drugging DNA Damage Repair Pathways for Trinucleotide Repeat Expansion Diseases

  2020cites this paper
• Rate of torque development and striatal shape in individuals with prodromal Huntington's disease

  2020cites this paper
• Cortical morphometry and neural dysfunction in Huntington’s disease: a review

  2020cites this paper
• The presymptomatic phase of amyotrophic lateral sclerosis: are we merely scratching the surface?

  2020cites this paper