Standard protocol approvals, registrations, and patient consents were obtained by the participating medical centers.
AVERAGE DATA IGOR PRO TRIAL
Key decision makers for each trial were contacted and asked to donate de-identified trial datasets. We describe the methods for creating the PRO-ACT database, provide a detailed description of its contents, summarize the baseline (trial entry) characteristics of the population and longitudinal clinical outcomes, and identify novel predictors of disease progression in this large cohort of people with ALS.Īll phase II and III ALS clinical trials completed between 19 with at least 80 subjects were eligible for inclusion in the PRO-ACT database ( figure 1). The database has been made publicly available with the goal of facilitating research that might leverage its remarkable statistical power for meaningful disease insights. Sixteen phase II and III ALS trials and one large observational study, conducted over the past 2 decades, are currently included, and this number is expected to increase.
This database represents the largest aggregation of ALS clinical trial data available. The Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database was designed to provide just such an opportunity. 3 Therefore, aggregation of studies is needed to allow enough statistical power to answer important questions about ALS natural history and clinical symptoms in order to overcome some of the barriers associated with drug development for orphan diseases. 1īecause ALS is a rare disease with an annual incidence of 2/100,000, 2 clinical trials have typically been relatively small, with the largest studies including fewer than 1,000 participants. People with ALS have progressive voluntary muscle weakness involving the arms, legs, speech, swallowing, and breathing. Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder that affects motor neurons in the brain and spinal cord.
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