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Poster: A Machine Learning ALS Survival Model Lacking Vital Capacity for use in Clinical Trials during the COVID-19 Pandemic

Presented at the 31st International Symposium on ALS/MND, December 9, 2020. Background: The ongoing COVID-19 pandemic has made it difficult in some clinical settings to measure vital capacity for the purpose of eligibility determination. We developed a machine...

Poster: Detectable Effect Cluster Analysis: A Novel Machine-Learning Subgroup Analysis Method for Drug Rescue

Presented at the 31st International Symposium on ALS/MND, December 9, 2020. Background: ALS drug development has been plagued by high clinical trial failure rates. Subgroup analysis is a key tool used to account for patient heterogeneity, but current methods fall...

Poster: Detectable Effect Cluster Analysis: A Novel Machine-Learning Based Clinical Trial Subgroup Analysis Tool

Presented at the 30th International Symposium on ALS/MND in Perth, Australia on December 4, 2019. Background: We previously developed machine-learning based ALS predictive models<sub>1-4</sub>, including a time to 50% expected vital capacity (VC50) model....

Poster: Estimate of an Acthar® Gel Treatment Effect in ALS Patients using Virtual Controls

Presented at the 30th International Symposium on ALS/MND in Perth, Australia on December 4, 2019. Background: Trial NCT01906658 was a small, 8-week randomized, open-label trial to evaluate the safety and tolerability of 4 Acthar® gel (repository corticotropin...
Poster: Detectable Effect Cluster Analysis: A Novel Machine Learning Based Clinical Trial Subgroup Analysis Tool

Poster: Detectable Effect Cluster Analysis: A Novel Machine Learning Based Clinical Trial Subgroup Analysis Tool

Presented at the 18th Annual NEALS Meeting in Clearwater, Florida, October 3rd, 2019. Background: We previously developed machine-learning based ALS predictive models, including a time to 50% expected vital capacity (VC50) model. Here we report on the use of the VC50...
Design and Analysis of a Clinical Trial Using Previous Trials as Historical Control

Design and Analysis of a Clinical Trial Using Previous Trials as Historical Control

David A. Schoenfeld, Dianne M. Finkelstein, Eric Macklin, Neta Zach, David L. Ennist, Albert A. Taylor, Nazem Atassi, The Pooled Resource Open-Access ALS Clinical Trials Consortium. DOI: 10.1177/1740774519858914 Published in Clinical Trials, on October, 2019.  This...
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